Raras
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Hipotireoidismo congênito
ORPHA:442CID-11 · 5A00.0PCDT · SUSDOENÇA RARA

Deficiência de hormônio tireoidiano presente desde o nascimento.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Deficiência de hormônio tireoidiano presente desde o nascimento.

Pesquisas ativas
9 ensaios
25 total registrados no ClinicalTrials.gov
Publicações científicas
4.286 artigos
Último publicado: 2026 Jun
Medicamentos
2 registrados
LEVOTHYROXINE SODIUM, LEVOTHYROXINE

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Ver detalhes, fases e interações →
LEVOTHYROXINE SODIUMLEVOTHYROXINE

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-5 / 10 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Europe
Início
Antenatal
+ neonatal
🏥
SUS: Cobertura parcialScore: 60%
PCDT disponívelTriagem neonatal (Fase 1)Centros em: PA, PR, SC, RS, ES +10
Você se identifica com essa condição?
O Raras está aqui pra te apoiar — com ou sem diagnóstico

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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

📏
Crescimento
38 sintomas
🧠
Neurológico
11 sintomas
🦴
Ossos e articulações
11 sintomas
👂
Ouvidos
8 sintomas
😀
Face
7 sintomas
👁️
Olhos
4 sintomas

+ 70 sintomas em outras categorias

Características mais comuns

Sonolência
Positividade do anticorpo anti-tireoperoxidase
Traços faciais grosseiros
Teste de descarga de perclorato positivo
Anormalidade da fisiologia da tireoide
Hiperbilirrubinemia conjugada
166sintomas
Sem dados (166)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 166 características clínicas mais associadas, ordenadas por frequência.

SonolênciaDrowsiness
Positividade do anticorpo anti-tireoperoxidaseAnti-thyroid peroxidase antibody positivity
Traços faciais grosseirosCoarse facial features
Teste de descarga de perclorato positivoPositive perchlorate discharge test
Anormalidade da fisiologia da tireoideAbnormality of thyroid physiology

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico4.286PubMed
Últimos 10 anos200publicações
Pico2025148 papers
Linha do tempo
2026Hoje · 2026🧪 1999Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Triagem neonatal (Teste do Pezinho)

👶
Teste: TSH neonatal em sangue seco
Fase 1 do PNTNTriagem nacionalimplemented_nationally
Incidência no Brasil: 1:3.500

A triagem neonatal permite diagnóstico precoce e início imediato do tratamento.

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

25 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive.

SLC26A4PendrinDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Sodium-independent transporter of chloride and iodide (PubMed:10192399, PubMed:11932316, PubMed:12107249, PubMed:16684826, PubMed:24051746). Mediates electroneutral chloride-bicarbonate, chloride-iodide and chloride-formate exchange with 1:1 stoichiometry (PubMed:10644529, PubMed:15155570, PubMed:24051746, PubMed:35601831). Mediates electroneutral iodide-bicarbonate exchange (By similarity)

LOCALIZAÇÃO

Cell membraneApical cell membrane

VIAS BIOLÓGICAS (1)
Inorganic anion exchange by SLC26 transporters
MECANISMO DE DOENÇA

Pendred syndrome

An autosomal recessive disorder characterized by congenital sensorineural hearing loss in association with thyroid goiter. The disorder may account for up to 10% of the cases of hereditary deafness. The deafness is most often associated with a Mondini cochlear defect. Deafness occurs early, starting at birth or during the first years of life. It is bilateral, sometimes asymmetrical, fluctuant and often progressive. Thyroid perturbations, such as thyroid goiter and/or hypothyroidism appear most commonly during adolescence, but they can be congenital or appear later.

EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
136.5 TPM
Brain Frontal Cortex BA9
3.3 TPM
Brain Anterior cingulate cortex BA24
3.0 TPM
Córtex cerebral
2.7 TPM
Rim - Córtex
2.2 TPM
OUTRAS DOENÇAS (5)
Pendred syndromeautosomal recessive nonsyndromic hearing loss 4thyroid hypoplasiaathyreosis
HGNC:8818UniProt:O43511
HESX1Homeobox expressed in ES cells 1Candidate gene tested inTolerante
FUNÇÃO

Required for the normal development of the forebrain, eyes and other anterior structures such as the olfactory placodes and pituitary gland. Possible transcriptional repressor. Binds to the palindromic PIII sequence, 5'-AGCTTGAGTCTAATTGAATTAACTGTAC-3'. HESX1 and PROP1 bind as heterodimers on this palindromic site, and, in vitro, HESX1 can antagonize PROP1 activation

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Septooptic dysplasia

A clinically heterogeneous disorder defined by any combination of optic nerve hypoplasia, pituitary gland hypoplasia with panhypopopituitarism, and midline abnormalities of the brain, including absence of the corpus callosum and septum pellucidum.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
9.7 TPM
Linfócitos
4.2 TPM
Pituitária
2.4 TPM
Nervo tibial
2.3 TPM
Tireoide
2.3 TPM
OUTRAS DOENÇAS (5)
septooptic dysplasiaKallmann syndromehypothyroidism due to deficient transcription factors involved in pituitary development or functionpituitary stalk interruption syndrome
HGNC:4877UniProt:Q9UBX0
POU1F1Pituitary-specific positive transcription factor 1Candidate gene tested inTolerante
FUNÇÃO

Transcription factor involved in the specification of the lactotrope, somatotrope, and thyrotrope phenotypes in the developing anterior pituitary. Specifically binds to the consensus sequence 5'-TAAAT-3'. Activates growth hormone and prolactin genes (PubMed:22010633, PubMed:26612202)

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Pituitary hormone deficiency, combined, 1

Combined pituitary hormone deficiency is defined as the impaired production of growth hormone and one or more of the other five anterior pituitary hormones. CPHD1 is characterized by pleiotropic deficiencies of growth hormone, prolactin and thyroid-stimulating hormone, while the production of adrenocorticotropic hormone, luteinizing hormone, and follicle-stimulating hormone are preserved. In infancy severe growth deficiency from birth as well as distinctive facial features with prominent forehead, marked midfacial hypoplasia with depressed nasal bridge, deep-set eyes, and a short nose with anteverted nostrils and hypoplastic pituitary gland by MRI examination can be seen. Some cases present with severe intellectual disability along with short stature.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
111.3 TPM
Cérebro - Hemisfério cerebelar
0.3 TPM
Cerebelo
0.2 TPM
Cervix Ectocervix
0.1 TPM
Fallopian Tube
0.1 TPM
OUTRAS DOENÇAS (4)
pituitary hormone deficiency, combined, 1hypothyroidism due to deficient transcription factors involved in pituitary development or functionisolated growth hormone deficiency type IIcombined pituitary hormone deficiencies, genetic form
HGNC:9210UniProt:P28069
LHX3LIM/homeobox protein Lhx3Candidate gene tested inTolerante
FUNÇÃO

Transcription factor. Recognizes and binds to the consensus sequence motif 5'-AATTAATTA-3' in the regulatory elements of target genes, such as glycoprotein hormones alpha chain CGA and visual system homeobox CHX10, positively modulating transcription; transcription can be co-activated by LDB2. Synergistically enhances transcription from the prolactin promoter in cooperation with POU1F1/Pit-1 (By similarity). Required for the establishment of the specialized cells of the pituitary gland and the n

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Regulation of expression of SLITs and ROBOs
MECANISMO DE DOENÇA

Pituitary hormone deficiency, combined, 3

Combined pituitary hormone deficiency is defined as the impaired production of growth hormone and one or more of the other five anterior pituitary hormones. CPHD3 is characterized by a complete deficit in all but one (adrenocorticotropin) anterior pituitary hormone and a rigid cervical spine leading to limited head rotation.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
89.7 TPM
Testículo
0.4 TPM
Skin Not Sun Exposed Suprapubic
0.1 TPM
Cerebelo
0.1 TPM
Cérebro - Hemisfério cerebelar
0.0 TPM
OUTRAS DOENÇAS (2)
non-acquired combined pituitary hormone deficiency with spine abnormalitieshypothyroidism due to deficient transcription factors involved in pituitary development or function
HGNC:6595UniProt:Q9UBR4
KCNJ10ATP-sensitive inward rectifier potassium channel 10Candidate gene tested inModerado
FUNÇÃO

May be responsible for potassium buffering action of glial cells in the brain (By similarity). Inward rectifier potassium channels are characterized by a greater tendency to allow potassium to flow into the cell rather than out of it (PubMed:8995301). Their voltage dependence is regulated by the concentration of extracellular potassium; as external potassium is raised, the voltage range of the channel opening shifts to more positive voltages (PubMed:8995301). The inward rectification is mainly d

LOCALIZAÇÃO

MembraneBasolateral cell membrane

VIAS BIOLÓGICAS (3)
Activation of G protein gated Potassium channelsInhibition of voltage gated Ca2+ channels via Gbeta/gamma subunitsPotassium transport channels
MECANISMO DE DOENÇA

Seizures, sensorineural deafness, ataxia, impaired intellectual development, and electrolyte imbalance

A complex disorder characterized by generalized seizures with onset in infancy, delayed psychomotor development, ataxia, sensorineural hearing loss, hypokalemia, metabolic alkalosis, and hypomagnesemia.

EXPRESSÃO TECIDUAL(Tecido-específico)
Brain Spinal cord cervical c-1
132.0 TPM
Brain Caudate basal ganglia
61.6 TPM
Substância negra
60.1 TPM
Brain Putamen basal ganglia
56.0 TPM
Córtex cerebral
49.6 TPM
OUTRAS DOENÇAS (4)
autosomal recessive nonsyndromic hearing loss 4EAST syndromePendred syndromeepisodic kinesigenic dyskinesia
HGNC:6256UniProt:P78508
FOXI1Forkhead box protein I1Candidate gene tested inTolerante
FUNÇÃO

Transcriptional activator required for the development of normal hearing, sense of balance and kidney function. Required for the expression of SLC26A4/PDS, JAG1 and COCH in a subset of epithelial cells and the development of the endolymphatic system in the inner ear. Also required for the expression of SLC4A1/AE1, SLC4A9/AE4, ATP6V1B1 and the differentiation of intercalated cells in the epithelium of distal renal tubules (By similarity)

LOCALIZAÇÃO

Nucleus

EXPRESSÃO TECIDUAL(Tecido-específico)
Rim - Córtex
32.3 TPM
Rim - Medula
24.0 TPM
Glândula salivar
8.9 TPM
Skin Sun Exposed Lower leg
4.0 TPM
Skin Not Sun Exposed Suprapubic
2.5 TPM
INTERAÇÕES PROTEICAS (4)
OUTRAS DOENÇAS (3)
autosomal recessive nonsyndromic hearing loss 4autosomal recessive distal renal tubular acidosisPendred syndrome
HGNC:3815UniProt:Q12951
PROP1Homeobox protein prophet of Pit-1Candidate gene tested inTolerante
FUNÇÃO

Possibly involved in the ontogenesis of pituitary gonadotropes, as well as somatotropes, lactotropes and caudomedial thyrotropes

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Pituitary hormone deficiency, combined, 2

Combined pituitary hormone deficiency is defined as the impaired production of growth hormone and one or more of the other five anterior pituitary hormones. CPHD2 is characterized by pleiotropic deficiencies of growth hormone, thyroid-stimulating hormone, follicle-stimulating hormone, luteinizing hormone, prolactin and adrenocorticotropic hormone.

EXPRESSÃO TECIDUAL(Baixa expressão)
Pituitária
2.0 TPM
Cerebelo
0.3 TPM
Cérebro - Hemisfério cerebelar
0.3 TPM
Testículo
0.1 TPM
Próstata
0.0 TPM
OUTRAS DOENÇAS (4)
pituitary hormone deficiency, combined, 2combined pituitary hormone deficiencies, genetic formhypothyroidism due to deficient transcription factors involved in pituitary development or functionpanhypopituitarism
HGNC:9455UniProt:O75360
TRHPro-thyrotropin-releasing hormoneCandidate gene tested inTolerante
FUNÇÃO

As a component of the hypothalamic-pituitary-thyroid axis, it controls the secretion of thyroid-stimulating hormone (TSH) and is involved in thyroid hormone synthesis regulation. It also operates as modulator of hair growth. It promotes hair-shaft elongation, prolongs the hair cycle growth phase (anagen) and antagonizes its termination (catagen) by TGFB2. It stimulates proliferation and inhibits apoptosis of hair matrix keratinocytes

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
G alpha (q) signalling eventsPeptide ligand-binding receptors
EXPRESSÃO TECIDUAL(Tecido-específico)
Hipotálamo
19.7 TPM
Cervix Endocervix
4.3 TPM
Ovário
3.7 TPM
Testículo
2.7 TPM
Brain Caudate basal ganglia
1.9 TPM
OUTRAS DOENÇAS (1)
isolated thyrotropin-releasing hormone deficiency
HGNC:12298UniProt:P20396
LHX4LIM/homeobox protein Lhx4Candidate gene tested inModerado
FUNÇÃO

May play a critical role in the development of respiratory control mechanisms and in the normal growth and maturation of the lung. Binds preferentially to methylated DNA (PubMed:28473536)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Regulation of expression of SLITs and ROBOs
MECANISMO DE DOENÇA

Pituitary hormone deficiency, combined, 4

Combined pituitary hormone deficiency is defined as the impaired production of growth hormone and one or more of the other five anterior pituitary hormones. CPHD4 is characterized by complete or partial deficiencies of growth hormone, thyroid-stimulating hormone, luteinizing hormone, follicle stimulating hormone and adrenocorticotropic hormone. Clinical features include short stature, cerebellar defects, and small sella turcica.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
5.2 TPM
Cerebelo
5.0 TPM
Cérebro - Hemisfério cerebelar
3.9 TPM
Pituitária
3.4 TPM
Tireoide
3.2 TPM
OUTRAS DOENÇAS (4)
short stature-pituitary and cerebellar defects-small sella turcica syndromehypothyroidism due to deficient transcription factors involved in pituitary development or functionpituitary stalk interruption syndromecombined pituitary hormone deficiencies, genetic form
HGNC:21734UniProt:Q969G2
IYDIodotyrosine deiodinase 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalyzes the dehalogenation of halotyrosines such as 3-bromo-L-tyrosine, 3-chloro-L-tyrosine, 3-iodo-L-tyrosine and 3,5-diiodo-L-tyrosine (PubMed:15289438, PubMed:18434651, PubMed:25395621, PubMed:28157283). During thyroid hormone biosynthesis, facilitates iodide salvage by catalysing the oxidative NADPH-dependent deiodination of the halogenated by-products of thyroid hormone production, monoiodotyrosine (L-MIT) and diiodotyrosine (L-DIT) (PubMed:15289438, PubMed:18434651). The scavanged iodide

LOCALIZAÇÃO

Cell membraneCytoplasmic vesicle membrane

VIAS BIOLÓGICAS (1)
Thyroxine biosynthesis
MECANISMO DE DOENÇA

Thyroid dyshormonogenesis 4

A disorder due to thyroid dyshormonogenesis, causing severe hypothyroidism, goiter, excessive levels of iodotyrosine in serum and urine, and variable mental deficits derived from unrecognized and untreated hypothyroidism.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
383.4 TPM
Fígado
3.7 TPM
Intestino delgado
3.2 TPM
Rim - Córtex
2.5 TPM
Cólon transverso
1.7 TPM
OUTRAS DOENÇAS (2)
thyroid dyshormonogenesis 4familial thyroid dyshormonogenesis
HGNC:21071UniProt:Q6PHW0
TBL1XF-box-like/WD repeat-containing protein TBL1XDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

F-box-like protein involved in the recruitment of the ubiquitin/19S proteasome complex to nuclear receptor-regulated transcription units (PubMed:14980219). Plays an essential role in transcription activation mediated by nuclear receptors. Probably acts as integral component of corepressor complexes that mediates the recruitment of the 19S proteasome complex, leading to the subsequent proteasomal degradation of transcription repressor complexes, thereby allowing cofactor exchange (PubMed:21240272

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (10)
PPARA activates gene expressionActivation of gene expression by SREBF (SREBP)Transcriptional regulation of white adipocyte differentiationRORA,B,C and NR1D1 (REV-ERBA) regulate gene expressionExpression of BMAL (ARNTL), CLOCK, and NPAS2
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 8

A form of central hypothyroidism, a disorder characterized by sub-optimal thyroid hormone secretion, due to insufficient stimulation by the thyroid stimulating hormone of an otherwise normal thyroid gland. It may be caused by congenital or acquired disorders of the pituitary gland or hypothalamus. CHNG8 is a congenital, X-linked, relatively mild form which may be accompanied by hearing loss in some patients.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
78.8 TPM
Cervix Endocervix
44.9 TPM
Cervix Ectocervix
38.7 TPM
Artéria tibial
38.7 TPM
Pituitária
37.0 TPM
OUTRAS DOENÇAS (1)
hypothyroidism, congenital, nongoitrous, 8
HGNC:HGNC:11585UniProt:O60907
TRHRThyrotropin-releasing hormone receptorDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Receptor for thyrotropin-releasing hormone (TRH). Upon ligand binding, this G-protein-coupled receptor triggers activation of the phosphatidylinositol (IP3)-calcium-protein kinase C (PKC) pathway

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (2)
G alpha (q) signalling eventsPeptide ligand-binding receptors
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 7

A form of central hypothyroidism, a disorder characterized by sub-optimal thyroid hormone secretion, due to insufficient stimulation by thyrotropin of an otherwise normal thyroid gland. It may be caused by congenital or acquired disorders of the pituitary gland or hypothalamus. CHNG7 is a congenital, autosomal recessive form characterized by normal-to-low T4 and normal-to-high thyrotropin levels, and reduced or absent pituitary responsiveness to thyrotropin-releasing hormone. Patients may exhibit short stature, growth retardation, and delayed bone age, as well as lethargy or fatigue.

EXPRESSÃO TECIDUAL(Baixa expressão)
Pituitária
0.9 TPM
Tireoide
0.1 TPM
Nervo tibial
0.1 TPM
Tecido adiposo
0.1 TPM
Músculo esquelético
0.0 TPM
OUTRAS DOENÇAS (1)
hypothyroidism, congenital, nongoitrous, 7
HGNC:12299UniProt:P34981
IGSF1Immunoglobulin superfamily member 1Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Seems to be a coreceptor in inhibin signaling, but seems not to be a high-affinity inhibin receptor. Antagonizes activin A signaling in the presence or absence of inhibin B (By similarity). Necessary to mediate a specific antagonistic effect of inhibin B on activin-stimulated transcription

LOCALIZAÇÃO

MembraneSecreted

MECANISMO DE DOENÇA

Hypothyroidism, central, and testicular enlargement

A disorder characterized by insufficient thyroid gland stimulation by thyroid stimulating hormone (TSH), resulting from hypothalamic and/or pituitary dysfunction. CHTE patients have delayed testosterone increase at puberty with normal testosterone levels in adulthood, normal testicular volume in childhood and enlarged testicles in adulthood.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
185.6 TPM
Hipotálamo
14.0 TPM
Nervo tibial
11.1 TPM
Testículo
9.8 TPM
Brain Nucleus accumbens basal ganglia
6.1 TPM
INTERAÇÕES PROTEICAS (1)
OUTRAS DOENÇAS (1)
X-linked central congenital hypothyroidism with late-onset testicular enlargement
HGNC:5948UniProt:Q8N6C5
DUOXA2Dual oxidase maturation factor 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Required for the maturation and transport of functional DUOX2 from the endoplasmic reticulum to the plasma membrane (PubMed:16651268). Recruits DUOX2 to the apical cell membrane (PubMed:39126279)

LOCALIZAÇÃO

Endoplasmic reticulum membraneApical cell membrane

VIAS BIOLÓGICAS (1)
Thyroxine biosynthesis
MECANISMO DE DOENÇA

Thyroid dyshormonogenesis 5

A disorder due to thyroid dyshormonogenesis, causing hypothyroidism, goiter, and variable mental deficits derived from unrecognized and untreated hypothyroidism.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
25.5 TPM
Vagina
7.4 TPM
Esôfago - Mucosa
7.3 TPM
Estômago
3.7 TPM
Skin Sun Exposed Lower leg
2.7 TPM
OUTRAS DOENÇAS (2)
thyroid dyshormonogenesis 5familial thyroid dyshormonogenesis
HGNC:32698UniProt:Q1HG44
TPOThyroid peroxidaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Iodination and coupling of the hormonogenic tyrosines in thyroglobulin to yield the thyroid hormones T(3) and T(4)

LOCALIZAÇÃO

MembraneCell surface

VIAS BIOLÓGICAS (1)
Thyroxine biosynthesis
VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
1180.0 TPM
Baço
19.9 TPM
Tecido adiposo
9.7 TPM
Mama
7.8 TPM
Esôfago - Junção
6.7 TPM
OUTRAS DOENÇAS (2)
thyroid dyshormonogenesis 2Afamilial thyroid dyshormonogenesis
HGNC:12015UniProt:P07202
SECISBP2Selenocysteine insertion sequence-binding protein 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

mRNA-binding protein that binds to the SECIS (selenocysteine insertion sequence) element present in the 3'-UTR of mRNAs encoding selenoproteins and facilitates the incorporation of the rare amino acid selenocysteine (PubMed:35709277). Insertion of selenocysteine at UGA codons is mediated by SECISBP2 and EEFSEC: SECISBP2 (1) specifically binds the SECIS sequence once the 80S ribosome encounters an in-frame UGA codon and (2) contacts the RPS27A/eS31 of the 40S ribosome before ribosome stalling (Pu

LOCALIZAÇÃO

NucleusMitochondrion

VIAS BIOLÓGICAS (1)
Selenocysteine synthesis
MECANISMO DE DOENÇA

Thyroid hormone metabolism, abnormal, 1

A disorder associated with a reduction in type II iodothyronine deiodinase activity.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
41.8 TPM
Ovário
40.0 TPM
Útero
32.8 TPM
Cervix Endocervix
31.7 TPM
Nervo tibial
30.9 TPM
OUTRAS DOENÇAS (1)
thyroid hormone metabolism, abnormal 1
HGNC:30972UniProt:Q96T21
DUOX2Dual oxidase 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Generates hydrogen peroxide which is required for the activity of thyroid peroxidase/TPO and lactoperoxidase/LPO (PubMed:15972824). Plays a role in thyroid hormone synthesis. Also required for lactoperoxidase-mediated antimicrobial defense at the surface of mucosa (PubMed:12824283). Synthesizes NAADP from its reduced NAADPH form which promotes Ca(2+) signaling during T cell activation (PubMed:34784249). May have its own peroxidase activity through its N-terminal peroxidase-like domain

LOCALIZAÇÃO

Apical cell membraneCell junction

VIAS BIOLÓGICAS (1)
Thyroxine biosynthesis
MECANISMO DE DOENÇA

Thyroid dyshormonogenesis 6

A disorder due to a defective conversion of accumulated iodide to organically bound iodine. The iodide organification defect can be partial or complete.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
124.8 TPM
Vagina
30.6 TPM
Esôfago - Mucosa
26.7 TPM
Estômago
9.8 TPM
Skin Not Sun Exposed Suprapubic
8.9 TPM
OUTRAS DOENÇAS (2)
thyroid dyshormonogenesis 6familial thyroid dyshormonogenesis
HGNC:13273UniProt:Q9NRD8
SLC5A5Sodium/iodide cotransporterDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Sodium:iodide symporter that mediates the transport of iodide into the thyroid gland (PubMed:12488351, PubMed:18372236, PubMed:18708479, PubMed:20797386, PubMed:31310151, PubMed:32084174, PubMed:8806637, PubMed:9329364). Can also mediate the transport of chlorate, thiocynate, nitrate and selenocynate (PubMed:12488351)

LOCALIZAÇÃO

Cell membraneCytoplasm

VIAS BIOLÓGICAS (2)
Thyroxine biosynthesisSLC-mediated transport of inorganic anions
MECANISMO DE DOENÇA

Thyroid dyshormonogenesis 1

A disorder characterized by the inability of the thyroid to maintain a concentration difference of readily exchangeable iodine between the plasma and the thyroid gland, leading to congenital hypothyroidism.

EXPRESSÃO TECIDUAL(Tecido-específico)
Estômago
6.2 TPM
Glândula salivar
4.6 TPM
Tireoide
4.4 TPM
Cerebelo
3.9 TPM
Cérebro - Hemisfério cerebelar
3.3 TPM
OUTRAS DOENÇAS (2)
thyroid dyshormonogenesis 1familial thyroid dyshormonogenesis
HGNC:11040UniProt:Q92911
IRS4Insulin receptor substrate 4Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Acts as an interface between multiple growth factor receptors possessing tyrosine kinase activity, such as insulin receptor, IGF1R and FGFR1, and a complex network of intracellular signaling molecules containing SH2 domains. Involved in the IGF1R mitogenic signaling pathway. Promotes the AKT1 signaling pathway and BAD phosphorylation during insulin stimulation without activation of RPS6KB1 or the inhibition of apoptosis. Interaction with GRB2 enhances insulin-stimulated mitogen-activated protein

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (1)
IRS-related events triggered by IGF1R
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 9

A form of central hypothyroidism, a disorder characterized by sub-optimal thyroid hormone secretion, due to insufficient stimulation by thyrotropin of an otherwise normal thyroid gland. It may be caused by congenital or acquired disorders of the pituitary gland or hypothalamus. CHNG9 is a congenital, X-linked recessive form. Patients have a small thyroid gland with low free T4 levels and inappropriately normal levels of thyrotropin.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
9.5 TPM
Ovário
2.7 TPM
Cervix Ectocervix
1.5 TPM
Tireoide
1.3 TPM
Hipotálamo
1.3 TPM
OUTRAS DOENÇAS (1)
hypothyroidism, congenital, nongoitrous, 9
HGNC:HGNC:6128UniProt:O14654
THRAThyroid hormone receptor alphaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Nuclear hormone receptor that can act as a repressor or activator of transcription. High affinity receptor for thyroid hormones, including triiodothyronine and thyroxine Does not bind thyroid hormone and functions as a weak dominant negative inhibitor of thyroid hormone action

LOCALIZAÇÃO

NucleusCytoplasm

VIAS BIOLÓGICAS (1)
Nuclear Receptor transcription pathway
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 6

A disease characterized by growth retardation, developmental retardation, skeletal dysplasia, borderline low thyroxine levels and high triiodothyronine levels. There is differential sensitivity to thyroid hormone action, with retention of hormone responsiveness in the hypothalamic pituitary axis and liver but skeletal, gastrointestinal, and myocardial resistance.

EXPRESSÃO TECIDUAL(Ubíquo)
Brain Nucleus accumbens basal ganglia
172.8 TPM
Cérebro - Hemisfério cerebelar
144.5 TPM
Cerebelo
126.3 TPM
Brain Caudate basal ganglia
110.4 TPM
Brain Anterior cingulate cortex BA24
109.7 TPM
OUTRAS DOENÇAS (2)
congenital nongoitrous hypothyroidism 6resistance to thyroid hormone due to a mutation in thyroid hormone receptor alpha
HGNC:11796UniProt:P10827
PAX8Paired box protein Pax-8Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor for the thyroid-specific expression of the genes exclusively expressed in the thyroid cell type, maintaining the functional differentiation of such cells

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (2)
Formation of the nephric ductFormation of intermediate mesoderm
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 2

A disease characterized by thyroid dysgenesis, the most frequent cause of congenital hypothyroidism, accounting for 85% of case. The thyroid gland can be completely absent (athyreosis), ectopically located and/or severely hypoplastic. Ectopic thyroid gland is the most frequent malformation, with thyroid tissue being found most often at the base of the tongue.

EXPRESSÃO TECIDUAL(Ubíquo)
Tireoide
1192.7 TPM
Rim - Medula
275.9 TPM
Rim - Córtex
170.0 TPM
Fallopian Tube
28.9 TPM
Glândula adrenal
5.5 TPM
OUTRAS DOENÇAS (5)
hypothyroidism, congenital, nongoitrous, 2thyroid hypoplasiaathyreosisdifferentiated thyroid carcinoma
HGNC:8622UniProt:Q06710
TSHRThyrotropin receptorDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Receptor for the thyroid-stimulating hormone (TSH) or thyrotropin (PubMed:11847099, PubMed:12045258). Also acts as a receptor for the heterodimeric glycoprotein hormone (GPHA2:GPHB5) or thyrostimulin (PubMed:12045258). TSHR is coupled to G(s) proteins and mediates the activation of adenylate cyclase (PubMed:11847099, PubMed:35940205, PubMed:35940204). This leads to the generation of cyclic adenosine monophosphate (cAMP), which in turn activates protein kinase A (PKA). PKA subsequently phosphoryl

LOCALIZAÇÃO

Cell membraneBasolateral cell membrane

VIAS BIOLÓGICAS (2)
G alpha (s) signalling eventsHormone ligand-binding receptors
EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
198.9 TPM
Fallopian Tube
0.9 TPM
Brain Caudate basal ganglia
0.5 TPM
Baço
0.5 TPM
Cervix Endocervix
0.4 TPM
OUTRAS DOENÇAS (5)
familial hyperthyroidism due to mutations in TSH receptorhypothyroidism due to TSH receptor mutationsfamilial gestational hyperthyroidismathyreosis
HGNC:12373UniProt:P16473
TSHBThyrotropin subunit betaDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Indispensable for the control of thyroid structure and metabolism

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (4)
G alpha (s) signalling eventsHormone ligand-binding receptorsThyroxine biosynthesisGlycoprotein hormones
MECANISMO DE DOENÇA

Hypothyroidism, congenital, non-goitrous, 4

A form of central hypothyroidism, a disorder characterized by insufficient stimulation by thyroid stimulating hormone of an otherwise normal thyroid gland. CHNG4 is an autosomal recessive form characterized by isolated thyrotropin deficiency that, if untreated, results in severe growth retardation and intellectual disability.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
634.8 TPM
Testículo
1.0 TPM
Útero
0.9 TPM
Artéria tibial
0.9 TPM
Cólon sigmoide
0.7 TPM
OUTRAS DOENÇAS (1)
isolated thyroid-stimulating hormone deficiency
HGNC:12372UniProt:P01222
NKX2-5Homeobox protein Nkx-2.5Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor required for the development of the heart and the spleen (PubMed:22560297). During heart development, acts as a transcriptional activator of NPPA/ANF in cooperation with GATA4 (By similarity). May cooperate with TBX2 to negatively modulate expression of NPPA/ANF in the atrioventricular canal (By similarity). Binds to the core DNA motif of NPPA promoter (PubMed:22849347, PubMed:26926761). Together with PBX1, required for spleen development through a mechanism that involves CD

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (3)
Physiological factorsYAP1- and WWTR1 (TAZ)-stimulated gene expressionCardiogenesis
MECANISMO DE DOENÇA

Atrial septal defect 7, with or without atrioventricular conduction defects

A congenital heart malformation characterized by incomplete closure of the wall between the atria resulting in blood flow from the left to the right atria, and atrioventricular conduction defects in some cases.

EXPRESSÃO TECIDUAL(Tecido-específico)
Coração - Átrio
113.8 TPM
Coração - Ventrículo esquerdo
108.3 TPM
Baço
44.1 TPM
Adipose Visceral Omentum
0.5 TPM
Testículo
0.3 TPM
OUTRAS DOENÇAS (15)
hypoplastic left heart syndrome 2tetralogy of fallotventricular septal defect 3hypothyroidism, congenital, nongoitrous, 5
HGNC:2488UniProt:P52952
TGThyroglobulinDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Acts as a substrate for the production of iodinated thyroid hormones thyroxine (T4) and triiodothyronine (T3) (PubMed:17532758, PubMed:32025030). The synthesis of T3 and T4 involves iodination of selected tyrosine residues of TG/thyroglobulin followed by their oxidative coupling in the thyroid follicle lumen (PubMed:32025030). Following TG re-internalization and lysosomal-mediated proteolysis, T3 and T4 are released from the polypeptide backbone leading to their secretion into the bloodstream (P

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
Pyrimidine salvagePurine salvage
MECANISMO DE DOENÇA

Thyroid dyshormonogenesis 3

A disorder due to thyroid dyshormonogenesis, causing large goiters of elastic and soft consistency in the majority of patients. Although the degree of thyroid dysfunction varies considerably among patients with defective thyroglobulin synthesis, patients usually have a relatively high serum free triiodothyronine (T3) concentration with disproportionately low free tetraiodothyronine (T4) level. The maintenance of relatively high free T3 levels prevents profound tissue hypothyroidism except in brain and pituitary, which are dependent on T4 supply, resulting in neurologic and intellectual defects in some cases.

EXPRESSÃO TECIDUAL(Tecido-específico)
Tireoide
7302.2 TPM
Testículo
3.8 TPM
Skin Sun Exposed Lower leg
3.3 TPM
Skin Not Sun Exposed Suprapubic
3.0 TPM
Próstata
2.3 TPM
OUTRAS DOENÇAS (3)
thyroid dyshormonogenesis 3familial thyroid dyshormonogenesisautoimmune thyroid disease, susceptibility to, 3
HGNC:11764UniProt:P01266

Medicamentos e terapias

LEVOTHYROXINE SODIUMPhase 4

Mecanismo: Thyroid hormone receptor agonist

LEVOTHYROXINEPhase 3

Mecanismo: Thyroid hormone receptor agonist

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

972 variantes patogênicas registradas no ClinVar.

🧬 SLC26A4: NM_000441.2(SLC26A4):c.1636A>G (p.Lys546Glu) ()
🧬 SLC26A4: NM_000441.2(SLC26A4):c.368dup (p.Ile124fs) ()
🧬 SLC26A4: NM_000441.2(SLC26A4):c.334C>G (p.Pro112Ala) ()
🧬 SLC26A4: NM_000441.2(SLC26A4):c.2006A>T (p.Asp669Val) ()
🧬 SLC26A4: NM_000441.2(SLC26A4):c.1634T>G (p.Val545Gly) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 561 variantes classificadas pelo ClinVar.

140
421
Patogênica (25.0%)
VUS (75.0%)
VARIANTES MAIS SIGNIFICATIVAS
PAX8: NM_003466.4(PAX8):c.268_270del (p.Glu90del) [Likely pathogenic]
TSHR: NM_000369.5(TSHR):c.497del (p.Thr165_Ser166insTer) [Pathogenic]
IGSF1: NM_001555.5(IGSF1):c.2036_2037insA (p.Thr680fs) [Likely pathogenic]
GLIS3: NM_001042413.2(GLIS3):c.2038C>T (p.Gln680Ter) [Likely pathogenic]
IGSF1: NM_001555.5(IGSF1):c.2816_2819dup (p.Tyr940Ter) [Likely pathogenic]

Vias biológicas (Reactome)

53 vias biológicas associadas aos genes desta condição.

Inorganic anion exchange by SLC26 transporters Defective SLC26A4 causes Pendred syndrome (PDS) Regulation of expression of SLITs and ROBOs Activation of G protein gated Potassium channels Potassium transport channels Inhibition of voltage gated Ca2+ channels via Gbeta/gamma subunits Peptide ligand-binding receptors G alpha (q) signalling events Thyroxine biosynthesis BMAL1:CLOCK,NPAS2 activates circadian expression PPARA activates gene expression NOTCH1 Intracellular Domain Regulates Transcription Transcriptional activation of mitochondrial biogenesis Activation of gene expression by SREBF (SREBP) Constitutive Signaling by NOTCH1 PEST Domain Mutants Constitutive Signaling by NOTCH1 HD+PEST Domain Mutants HDACs deacetylate histones Notch-HLH transcription pathway Transcriptional regulation of white adipocyte differentiation Regulation of lipid metabolism by PPARalpha Loss of MECP2 binding ability to the NCoR/SMRT complex Regulation of MECP2 expression and activity NR1H3 & NR1H2 regulate gene expression linked to cholesterol transport and efflux HCMV Early Events Cytoprotection by HMOX1 Heme signaling MLL4 and MLL3 complexes regulate expression of PPARG target genes in adipogenesis and hepatic steatosis Expression of BMAL (ARNTL), CLOCK, and NPAS2 RORA,B,C and NR1D1 (REV-ERBA) regulate gene expression Selenocysteine synthesis Defective SLC5A5 causes thyroid dyshormonogenesis 1 (TDH1) SLC-mediated transport of inorganic anions IRS-related events triggered by IGF1R Nuclear Receptor transcription pathway SUMOylation of intracellular receptors Formation of intermediate mesoderm Formation of the nephric duct Hormone ligand-binding receptors G alpha (s) signalling events Glycoprotein hormones YAP1- and WWTR1 (TAZ)-stimulated gene expression Physiological factors Cardiogenesis Response of EIF2AK4 (GCN2) to amino acid deficiency Regulation of localization of FOXO transcription factors Chylomicron assembly Defective Base Excision Repair Associated with NEIL1 Nuclear signaling by ERBB4 Signaling by high-kinase activity BRAF mutants GABA receptor activation AKT-mediated inactivation of FOXO1A Regulation of MITF-M-dependent genes involved in lysosome biogenesis and autophagy Defective MUTYH substrate processing

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Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Hipotireoidismo congênito

Centros de Referência SUS

24 centros habilitados pelo SUS para Hipotireoidismo congênito

Centros para Hipotireoidismo congênito

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Hospital Universitário Prof. Edgard Santos (HUPES)

R. Dr. Augusto Viana, s/n - Canela, Salvador - BA, 40110-060 · CNES 0003808

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Infantil Albert Sabin

R. Tertuliano Sales, 544 - Vila União, Fortaleza - CE, 60410-794 · CNES 2407876

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Rota
Anomalias CongênitasDeficiência Intelectual

Hospital de Apoio de Brasília (HAB)

AENW 3 Lote A Setor Noroeste - Plano Piloto, Brasília - DF, 70684-831 · CNES 0010456

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Estadual Infantil e Maternidade Alzir Bernardino Alves (HIABA)

Av. Min. Salgado Filho, 918 - Soteco, Vila Velha - ES, 29106-010 · CNES 6631207

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Rota
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Hospital das Clínicas da UFG

Rua 235 QD. 68 Lote Área, Nº 285, s/nº - Setor Leste Universitário, Goiânia - GO, 74605-050 · CNES 2338424

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Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Universitário da UFJF

R. Catulo Breviglieri, Bairro - s/n - Santa Catarina, Juiz de Fora - MG, 36036-110 · CNES 2297442

Atenção Especializada

Rota
Anomalias Congênitas

Hospital das Clínicas da UFMG

Av. Prof. Alfredo Balena, 110 - Santa Efigênia, Belo Horizonte - MG, 30130-100 · CNES 2280167

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Julio Müller (HUJM)

R. Luis Philippe Pereira Leite, s/n - Alvorada, Cuiabá - MT, 78048-902 · CNES 2726092

Atenção Especializada

Rota
Anomalias Congênitas

Hospital Universitário João de Barros Barreto

R. dos Mundurucus, 4487 - Guamá, Belém - PA, 66073-000 · CNES 2337878

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Lauro Wanderley (HULW)

R. Tabeliao Estanislau Eloy, 585 - Castelo Branco, João Pessoa - PB, 58050-585 · CNES 0002470

Atenção Especializada

Rota
Anomalias Congênitas

Instituto de Medicina Integral Prof. Fernando Figueira (IMIP)

R. dos Coelhos, 300 - Boa Vista, Recife - PE, 50070-902 · CNES 0000647

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Pequeno Príncipe

R. Des. Motta, 1070 - Água Verde, Curitiba - PR, 80250-060 · CNES 3143805

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Rota
Anomalias CongênitasDeficiência Intelectual

Hospital Universitário Regional de Maringá (HUM)

Av. Mandacaru, 1590 - Parque das Laranjeiras, Maringá - PR, 87083-240 · CNES 2216108

Atenção Especializada

Rota
Anomalias Congênitas

Hospital de Clínicas da UFPR

R. Gen. Carneiro, 181 - Alto da Glória, Curitiba - PR, 80060-900 · CNES 2364980

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Pedro Ernesto (HUPE-UERJ)

Blvd. 28 de Setembro, 77 - Vila Isabel, Rio de Janeiro - RJ, 20551-030 · CNES 2280221

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Rota
Anomalias CongênitasErros Inatos do Metabolismo

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira (IFF/Fiocruz)

Av. Rui Barbosa, 716 - Flamengo, Rio de Janeiro - RJ, 22250-020 · CNES 2269988

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital São Lucas da PUCRS

Av. Ipiranga, 6690 - Jardim Botânico, Porto Alegre - RS, 90610-000 · CNES 2232928

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital de Clínicas de Porto Alegre (HCPA)

Rua Ramiro Barcelos, 2350 Bloco A - Av. Protásio Alves, 211 - Bloco B e C - Santa Cecília, Porto Alegre - RS, 90035-903 · CNES 2237601

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Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário da UFSC (HU-UFSC)

R. Profa. Maria Flora Pausewang - Trindade, Florianópolis - SC, 88036-800 · CNES 2560356

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Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital das Clínicas da FMUSP

R. Dr. Ovídio Pires de Campos, 225 - Cerqueira César, São Paulo - SP, 05403-010 · CNES 2077485

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Hospital de Base de São José do Rio Preto

Av. Brg. Faria Lima, 5544 - Vila Sao Jose, São José do Rio Preto - SP, 15090-000 · CNES 2079798

Atenção Especializada

Rota
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Hospital de Clínicas da UNICAMP

R. Vital Brasil, 251 - Cidade Universitária, Campinas - SP, 13083-888 · CNES 2748223

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Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas de Ribeirão Preto (HCRP-USP)

R. Ten. Catão Roxo, 3900 - Vila Monte Alegre, Ribeirão Preto - SP, 14015-010 · CNES 2082187

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UNIFESP / Hospital São Paulo

R. Napoleão de Barros, 715 - Vila Clementino, São Paulo - SP, 04024-002 · CNES 2688689

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Rota
Anomalias CongênitasErros Inatos do Metabolismo
Sobre os centros SUS: Estes centros são habilitados pelo Ministério da Saúde como Serviços de Referência em Doenças Raras ou Serviços de Atenção Especializada. O atendimento é pelo SUS, com encaminhamento da rede de atenção básica.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

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Publicações mais relevantes

Timeline de publicações
1.636 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 1.636

#1

A rare case report of familial glucocorticoid deficiency type 4 (GCCD4) with dilated cardiomyopathy: a 3-year follow-up study.

Translational pediatrics2026 Feb 28

Familial glucocorticoid deficiency type 4 (GCCD4), caused by nicotinamide nucleotide transhydrogenase (NNT) gene mutations, represents a rare multisystem disorder with poorly characterized cardiac manifestations. A 12-year-old female presented with recurrent fatigue, syncope, and severe arrhythmias. After multiple misdiagnoses [including dilated cardiomyopathy, fulminant myocarditis, and long QT syndrome (LQTS)], comprehensive evaluation revealed hypocortisolism (<1.0 µg/dL), markedly elevated adrenocorticotropic hormone (ACTH) (>1,250 pg/mL), and hypothyroidism. Genetic testing identified compound heterozygous NNT mutations (c.639_640insC/p.Val214Argfs*32 and c.2764C>T/p.Arg922*). Comprehensive whole-exome sequencing (WES) ruled out pathogenic variants in genes associated with isolated cardiomyopathy or congenital hypothyroidism. Cardiac assessment showed left ventricular dilation [left ventricular end-diastolic diameter (LVEDD) 5.14 cm], reduced left ventricular ejection fraction (LVEF) 53%, and corrected QT interval (QTc) prolongation (559 ms) with torsades de pointes. The patient was managed with hormone replacement (hydrocortisone 8.8→16.1 mg/m2/day; levothyroxine 16.7 µg/day) and cardioprotective therapy (propranolol, captopril, coenzyme Q10), resulting in significant improvement at 3-year follow-up: ejection fraction (EF) normalized to 68%, thyroid function recovered permitting levothyroxine discontinuation, hyperpigmentation resolved completely, and no arrhythmias recurred. This case underscores GCCD4 as a differential diagnosis in pediatric cardiomyopathy with arrhythmias. Multidisciplinary collaboration and early genetic testing are critical for diagnosis. Individualized glucocorticoid dosing combined with cardiac support enables favorable outcomes.

#2

Predicting Pathogenicity of TSHR Missense Variants of Uncertain Significance: An Integrative Computational Study.

International journal of molecular sciences2026 Feb 06

Pathogenic variants in the thyroid-stimulating hormone receptor gene (TSHR) contribute to a wide spectrum of thyroid dysfunctions, ranging from congenital hypothyroidism to thyrotropin resistance. With the advancement of bioinformatics algorithms for variant effect prediction, assessing the pathogenic potential of variants has become increasingly important. This study aimed to investigate the pathogenic effects of TSHR variants classified as variants of uncertain significance (VUSs) in the gnomAD v4.1.0 database. TSHR variants listed in gnomAD v4.1.0 were retrieved and filtered to select missense VUSs based on ClinVar classifications. Multiple bioinformatics tools were used to assess the secondary and three-dimensional structures of the TSHR, as well as protein stability, evolutionary conservation, and molecular dynamics simulations. A total of 2760 TSHR variants were found in gnomAD v4.1.0, including 75 frameshifts, 80 splice-sites, 265 in the 3' and 5' untranslated regions, 422 synonymous, 892 others, and 1026 missense variants. Among these, 68 missense VUSs were identified and selected for bioinformatics analysis. Three variants (p.Cys29Trp, p.Leu57Pro, and p.Phe97Ser) were consistently predicted to be pathogenic by all the bioinformatics tools used. All three variants were located within the leucin-rich repeat domain extracellular region of the TSHR and within a highly conserved region across species. Molecular dynamics simulations for mutant proteins (p.Cys29Trp, p.Leu57Pro, and p.Phe97Ser) reveal structural instability in comparison to the wild protein. Comprehensive bioinformatics analysis revealed that three TSHR missense VUSs exhibited pathogenic potential. These variants may contribute to thyroid dysfunction by affecting the receptor's structural and signalling integrity.

#3

Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.

Journal of tropical pediatrics2026 Jan 02

Neonatal screening has not been demanded by pediatricians nor parents in India because of limited resources, inadequate health information, early hospital discharges, a large rate of deliveries at home, and challenges with follow-up of results. This study was conducted based on the laboratory e-records from the Newborn Screening Unit, where Dried Blood Spot-based analysis was performed for G6PD deficiency, congenital adrenal hyperplasia, neonatal hypothyroidism, biotinidase deficiency, and galactosemia. The total study population comprised 3210 neonates. Overall, 329 children (10.2%) had some form of inherited disorder, and 12 (0.4%) suffered from two disorders concomitantly. The highest number of children were suffering from partial biotinidase deficiency (4.5%), followed by G6PD deficiency (2.7%). There was no significant difference in the levels of measured analytes in males and females, except for 17-OHP, which was significantly higher in males. Compared with females, males were more likely to have G6PD and biotinidase deficiency. Our study provides a snapshot of five inherited disorders previously unexamined by neonatal screening in Eastern India. We found a high prevalence of G6PD deficiency and other substantive neonatal conditions. Now that the significant prevalence of these disorders is known, stakeholders should work with policy-makers to institute national-level screening to determine their distribution in the broader population and address them in affected groups. Further studies are needed to identify effective implementation strategies, overcome financial and logistical barriers to an NBS program, and assess whether such a program can reach and benefit all communities, especially the most vulnerable.

#4

Perinatal Factors Associated With Congenital Hypothyroidism in Very Low Birth Weight Infants: A Retrospective Cohort Study.

Clinical pediatrics2026 Jan

This retrospective cohort study analyzed perinatal risk factors for congenital hypothyroidism (CH) in 1073 very low birth weight (VLBW) infants (<1500 g) admitted to a neonatal intensive care unit. The overall CH incidence was 6.0% (65 cases), with significantly higher rates in extremely low birth weight (ELBW) infants (11.1%) and extremely preterm infants <28 weeks' gestation (11.3%). Multivariate analysis identified maternal thyroid disease (odds ratio [OR]: 2.12, 95% confidence interval [CI]: 1.10-4.12) and patent ductus arteriosus (OR: 2.25, 95% CI: 1.33-3.79) as independent risk factors, while higher birth weight showed a marginally protective effect (OR 0.998, 95% CI: 0.997-1.000). The elevated CH incidence among extremely preterm infants and those with lower birth weights highlights the needs for dynamic thyroid monitoring in VLBW populations, particularly when maternal thyroid disease is present. These findings justify risk-stratified surveillance protocols to facilitate early detection and optimize clinical management in high-risk neonates.

#5

Congenital hypothyroidism and thyroid dysgenesis: importance of newborn bloodspot screening in Ireland.

Irish journal of medical science2026 Feb 14

Publicações recentes

Ver todas no PubMed

📚 EuropePMC2.636 artigos no totalmostrando 196

2026

Hearing assessment of Egyptian children with permanent congenital hypothyroidism: A single-center experience.

International journal of pediatric otorhinolaryngology
2026

Birth Prevalence of Endocrine-Metabolic Disorders Detected by Newborn Screening Test in Pune (India) Population.

Indian journal of clinical biochemistry : IJCB
2026

A rare case report of familial glucocorticoid deficiency type 4 (GCCD4) with dilated cardiomyopathy: a 3-year follow-up study.

Translational pediatrics
2026

Rapid peptide analysis in dried bloodspots to identify novel markers for newborn screening for congenital hypothyroidism.

Scientific reports
2026

Finnish-Enriched SLC26A7 Variant in Congenital Hypothyroidism: Clinical Spectrum, Thyroid Histopathology, and Expression Analysis.

Thyroid : official journal of the American Thyroid Association
2026

Early prediction of transient versus permanent congenital hypothyroidism: a retrospective cohort study.

Annals of pediatric endocrinology &amp; metabolism
2026

Analysis of epidemiological characteristics and associated factors of congenital hypothyroidism in Henan Province.

Scientific reports
2026

Newborn screening for congenital hypothyroidism in France: a study of current professional practices.

European thyroid journal
2026

Structural and catalytic consequences of active-site vs. distal mutations in human dehalogenase: insights from molecular dynamics simulations.

Physical chemistry chemical physics : PCCP
2026

Methyltransferase like 3 promotes thyroid folliculogenesis via coordinating cell differentiation and polarization.

Journal of translational internal medicine
2026

Integrated Newborn Screening in Nigeria: The Way Forward, A Workshop Report.

International journal of neonatal screening
2026

Association between prenatal exposure to residential pyrethroid insecticides and congenital hypothyroidism using propensity score matching.

Scientific reports
2026

Case report of breastfeeding after maternal iodine contrast: neonatal hypothyroidism revealing an underlying congenital disorder.

International breastfeeding journal
2026

Congenital hypothyroidism and thyroid dysgenesis: importance of newborn bloodspot screening in Ireland.

Irish journal of medical science
2025

Effect of timely diagnosis and treatment on growth and body proportionality of children with congenital hypothyroidism.

Frontiers in endocrinology
2026

Predicting Pathogenicity of TSHR Missense Variants of Uncertain Significance: An Integrative Computational Study.

International journal of molecular sciences
2026

Co-existence of Congenital Hypothyroidism (CH) and TBG-Excess in a Boy Causing Simultaneous Elevation in Thyroid Stimulating Hormone (TSH) and Thyroxine (T4) Levels: First Report from India and Review of the Literature.

Indian journal of clinical biochemistry : IJCB
2025

Evaluating cardiac echocardiographic changes with levothyroxine in hypothyroid patients: a systematic review and meta-analysis.

Frontiers in endocrinology
2026

Comparative Analysis of Hyperbilirubinemia Between Congenital Hypothyroidism and Euthyroidism in Neonates in Rawalpindi, Pakistan.

Cureus
2026

Hypothyroidism-Etiologies, Evaluation, and Emergency Care.

Endocrinology and metabolism clinics of North America
2026

A Novel PTEN Frameshift Variant in a Child With Autism Spectrum Disorder and Macrocephaly: A Case Report.

Cureus
2026

Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.

Journal of tropical pediatrics
2026

DYRK1A Expression and Thyroid Dysfunction in Subjects With Down Syndrome.

Clinical endocrinology
2026

Air pollution and risk of autoimmune thyroid diseases in children and adolescents: Findings from a nationwide cohort study in Korea.

Ecotoxicology and environmental safety
2026

Predicting congenital hypothyroidism in newborns with complex risk profiles by using thyroid-stimulating hormone variations across serial dried blood spots.

Clinical chemistry and laboratory medicine
2026

Beyond the Gut: Thyroxine Malabsorption, Pharmacokinetics, and Delivery.

The Journal of clinical endocrinology and metabolism
2026

Delayed Diagnosis of Ectopic Thyroid Due to Ignored Borderline Result of Newborn Screening for Congenital Hypothyroidism.

The American journal of case reports
2026

Preterm heart failure and refractory lactic acidosis caused by congenital hypothyroidism: A case report and review of literature.

World journal of clinical cases
2026

Integrating next-generation sequencing with biochemical screening to optimize first-tier newborn screening systems.

Human genomics
2025

rs9789446 genotype as susceptibility biomarkers for congenital hypothyroidism based on population and family validation.

Frontiers in genetics
2025

Transient congenital hypothyroidism with normal size thyroid caused by maternal thyrotropin receptor antibodies: a case report and literature review.

BMC pregnancy and childbirth
2025

Enhancing TSH-based congenital hypothyroidism screening using machine learning and resampling algorithms.

BMC medical informatics and decision making
2025

[Interpretation of the 2025 "Guidelines for the diagnosis and treatment of congenital hypothyroidism"].

Zhonghua yi xue za zhi
2025

Concomitant Mutations in the Thyroglobulin and SLC26A4 Genes Leading to Fetal Goiter and Congenital Hypothyroidism in a Patient With Pendred Syndrome.

Case reports in endocrinology
2025

Predictive Factors of Transient Congenital Hypothyroidism among Filipino Children: A Retrospective Study.

Acta medica Philippina
2026

Is the tracheal index useful for evaluating the thyroid in infants suspected of hypothyroidism?

Journal of pediatric endocrinology &amp; metabolism : JPEM
2026

Investigation of the Effects of Hypothyroidism Duration on Hearing Function: An Experimental Animal Study.

The Annals of otology, rhinology, and laryngology
2025

Revealing the genotype-phenotype correlations of congenital hypothyroidism in Yunnan Province, Southwest China.

Frontiers in endocrinology
2025

EndoCompass Project: Research Roadmap for Thyroid Endocrinology.

Hormone research in paediatrics
2026

Prevalence of congenital hypothyroidism in infants of mothers with hypothyroidism: a meta-analysis.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Molecular Genetics of Primary Congenital Hypothyroidism: Established and Emerging Contributors to Thyroid Dysgenesis.

International journal of molecular sciences
2025

Universal Decentralized Cord Blood TSH Screening Should Be Offered as Routine Delivery Care in Limited-Resource Settings.

International journal of neonatal screening
2025

Celebrating 50 Years of Nationwide Newborn Screening in Hungary-Review, Current Situation, and Future Directions.

International journal of neonatal screening
2025

Variation spectra in mild isolated hyperthyrotropinemia: pilot cohort and systematic review.

Frontiers in endocrinology
2025

Increasing prevalence of congenital hypothyroidism emerges as a growing concern in Jordan.

World journal of clinical pediatrics
2025

The herculean illusion: congenital hypothyroidism masquerading as muscular dystrophy.

Acta neurologica Belgica
2025

Tapering Levothyroxine Dose for Intra-Amniotic Infusion in the Antenatal Treatment of Fetal Goiter: A Case Report.

Case reports in obstetrics and gynecology
2025

Case Report: Hydroxychloroquine in an infant with NKX2-1-associated interstitial lung disease.

Frontiers in pediatrics
2025

Genetic Rescue of a Subset of Thyroid Follicular Cells Restores Thyroid Function in Dyshormonogenic Duoxa-/- Mice.

Thyroid : official journal of the American Thyroid Association
2025

Identification of hub genes in congenital hypothyroidism and construction of the associated immune regulatory network.

Frontiers in immunology
2025

Is there are relationship between polymorphisms TSHR gene frequencies and genetic ancestry markers in patients with Primary Congenital Hypothyroidism?

Genetics and molecular biology
2025

Correlation of Genotype-Phenotype of Congenital Hypothyroidism Cohort Diagnosed by Newborn Screening: A Long-Term Observational Study.

International journal of neonatal screening
2025

Congenital Hypothyroidism: Moving Ahead, but a Long Way Still to Go.

International journal of neonatal screening
2025

ITCH Deficiency Causing Immunodeficiency and Immune Dysregulation.

Pediatrics
2025

Congenital Glaucoma in Patients with Neonatal Diabetes and Congenital Hypothyroidism (NDH) Syndrome.

Klinische Monatsblatter fur Augenheilkunde
2025

Challenge faced in diagnosis and treatment of congenital hypothyroidism presenting with isosexual puberty and bilateral ovarian mass in low and middle income country: a case report.

BMC endocrine disorders
2025

Prematurity Appears to Be the Main Factor for Transient Congenital Hypothyroidism in Greece, a Recently Iodine-Replete Country.

Nutrients
2025

Term birth and levothyroxine dosage are significant factors associated with permanent congenital hypothyroidism: experience from a medical center in Taiwan.

BMC pediatrics
2026

Effects of iodine supplementation on thyroid function in children with mild congenital hypothyroidism and non-autoimmune subclinical hypothyroidism.

Journal of endocrinological investigation
2025

Severe Undiagnosed Congenital Hypothyroidism Due to Missed Newborn Screening.

JCEM case reports
2025

Impact of Maternal Obesity on Neonatal TSH Levels: A Prospective Study on the Influence of BMI.

American journal of perinatology
2025

Frequency of Congenital Hypothyroidism and Radiographic Assessment of Knee Epiphysis in Newborn.

Mymensingh medical journal : MMJ
2025

Congenital hypothyroidism in two children affected by Sotos syndrome: a simple association?

Italian journal of pediatrics
2025

Initial Levothyroxine Dose and Neurodevelopmental Outcomes in Children with Congenital Hypothyroidism.

Indian journal of pediatrics
2026

Functional Domain Mapping of TPO: Insights From 6 Variants in Sudanese Kindreds With Congenital Hypothyroidism.

The Journal of clinical endocrinology and metabolism
2025

Corrigendum to: Clinical Outcomes of Congenital Hypothyroidism Due to DUOX2 Biallelic Mutations after Levothyroxine Withdrawal.

Thyroid : official journal of the American Thyroid Association
2026

Newborn screening for rare diseases: expanding the paradigm in the genomic era.

Journal of perinatal medicine
2025

Altered carnitine-acylcarnitine profiles in levothyroxine-treated congenital hypothyroid patients with fatigue: An LC-MS/MS-based study from Bangladesh.

PloS one
2025

Congenital Hypothyroidism Dysregulates TRPC6 to Mediate Abnormal Dendritic Spine Growth of Hippocampal Neurons.

CNS neuroscience &amp; therapeutics
2026

Catch-Up Growth in Children With Congenital Hypothyroidism on Thyroxine Therapy: A Retrospective Study.

Clinical endocrinology
2025

Airway compression in a neonate with thyroid dyshormonogenesis due to SLC26A7 defect.

European journal of endocrinology
2025

The Burden of Congenital Hypothyroidism Without Newborn Screening: Clinical and Cognitive Findings from a Multicenter Study in Algeria.

International journal of neonatal screening
2026

Permanent or Transient Congenital Hypothyroidism: A Diagnostic Dilemma.

Acta paediatrica (Oslo, Norway : 1992)
2025

Epidemiologic and clinical aspects of congenital hypothyroidism in Côte d'Ivoire, a country without systematic screening at birth.

Annales d'endocrinologie
2025

Prevalence of pendrin defects in sudanese families with congenital hypothyroidism.

Endocrine
2025

Improving Clinical Care for Children with Congenital Hypothyroidism.

Pediatric quality &amp; safety
2025

Congenital Hypothyroidism: Long-Term Growth and Intellectual Outcomes With a Lower Initial Levothyroxine Dose.

Clinical endocrinology
2026

Effects of neonatal hypothyroidism on testicular development and undifferentiated spermatogonia in prepubertal rats.

Andrology
2025

Clinical and genetic characterization of Weaver syndrome: A case report of an EZH2 mutation and review of the literature.

Medicine
2025

Clinical Outcomes of Congenital Hypothyroidism Due to DUOX2 Biallelic Mutations after Levothyroxine Withdrawal.

Thyroid : official journal of the American Thyroid Association
2025

Commentary on &quot;Predictive factors of permanent versus transient congenital hypothyroidism: a pragmatic cohort study&quot.

Annals of pediatric endocrinology &amp; metabolism
2025

Quality of life in patients with overt hypothyroidism: a systematic review.

European journal of endocrinology
2025

Association between Human Leukocyte Antigen Alleles and Endocrine Disorders in a Cohort of 895 Patients from Russian Clinical Population.

Biochemistry. Biokhimiia
2026

Perinatal Factors Associated With Congenital Hypothyroidism in Very Low Birth Weight Infants: A Retrospective Cohort Study.

Clinical pediatrics
2025

Primary congenital hypothyroidism: a clinical review.

Frontiers in endocrinology
2025

Is It Time to Expand Newborn Screening for Congenital Hypothyroidism to Other Rare Thyroid Diseases?

International journal of neonatal screening
2025

Incidence of Congenital Hypothyroidism Is Increasing in Chile.

International journal of neonatal screening
2025

A Case Report of Tissue Mosaicism in 45,X0/46,XY: Diagnostic Complexity in a Newborn with Ambiguous Genitalia.

Reports (MDPI)
2025

Newborn Screening of the Endocrine System: Best Practices for Evaluation of Hypothyroidism and Congenital Adrenal Hyperplasia.

Clinics in perinatology
2025

Dominant-negative effects of Weaver syndrome-associated EZH2 variants.

Genes &amp; development
2025

Timing of adiposity rebound in children with congenital hypothyroidism diagnosed by newborn screening and treated with Levothyroxine.

Italian journal of pediatrics
2025

Differentiating transient and permanent congenital hypothyroidism: predictive clues from Istanbul, Türkiye.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Meta-analysis of the effects of levothyroxine therapy for subclinical hypothyroidism during pregnancy on offspring outcomes.

Frontiers in pediatrics
2025

Implementation of Neonatal Screening Program for Congenital Hypothyroidism in Eastern Morocco.

International journal of neonatal screening
2025

Associational study of neonatal hearing screening results and common metabolic disorders.

International journal of pediatric otorhinolaryngology
2025

50 YEARS OF NEWBORN SCREENING FOR CONGENITAL HYPOTHYROIDISM: EVOLUTION OF INSIGHTS IN ETIOLOGY, DIAGNOSIS AND MANAGEMENT: Transient or permanent congenital hypothyroidism: from milestones to current and future perspectives.

European thyroid journal
2025

Triple Ectopic Thyroid Gland in a 4-Year-Old Child.

AACE endocrinology and diabetes
2025

Impact of Maternal and Neonatal Factors on Thyroid-Stimulating Hormone Concentrations in Newborns in Eastern Morocco.

Cureus
2025

Screening of 50,539 newborns for congenital hypothyroidism: optimization of TSH cut-off values and seasonal impact in clinical practice.

Frontiers in endocrinology
2025

Predicting variables associated with diagnostic reevaluation of transient congenital hypothyroidism.

Annals of pediatric endocrinology &amp; metabolism
2025

Reconsidering the timing of levothyroxine discontinuation in children with congenital hypothyroidism: insights from a Korean Cohort Study - commentary on "Predicting variables associated with diagnostic reevaluation of transient congenital hypothyroidism".

Annals of pediatric endocrinology &amp; metabolism
2025

Iodine and Hypothyroidism.

Endocrine, metabolic &amp; immune disorders drug targets
2025

Early Mortalities From Inborn Errors of Metabolism Detected By Selective Screening in Malaysia.

Sage open pediatrics
2025

Diffuse infantile hepatic haemangioma and consumptive hypothyroidism: a clinical case with anaemia that raises suspicion.

Italian journal of pediatrics
2025

Congenital glaucoma associated with high hyperopia, an ophthalmic phenotypical manifestation for GLIS3 deletion: case report and review of literature.

Ophthalmic genetics
2025

50 YEARS OF NEWBORN SCREENING FOR CONGENITAL HYPOTHYROIDISM: EVOLUTION OF INSIGHTS IN ETIOLOGY, DIAGNOSIS AND MANAGEMENT: Management during pregnancy and long-term outcomes of adult patients with congenital hypothyroidism.

European thyroid journal
2025

Molecular and clinical characteristics of pediatric patients with primary congenital hypothyroidism: novel genetic variants and the genotype-phenotype association.

Clinica chimica acta; international journal of clinical chemistry
2025

Large-scale screening and functional study of DUOXA2 variant in 599 Chinese patients with congenital hypothyroidism.

European journal of endocrinology
2025

Thyroid function profiles in small versus appropriate for gestational age stable neonates with implications for screening cutoffs: a retrospective cross-sectional study.

The journal of maternal-fetal &amp; neonatal medicine : the official journal of the European Association of Perinatal Medicine, the Federation of Asia and Oceania Perinatal Societies, the International Society of Perinatal Obstetricians
2025

Delayed Thyrotropin Rise in Preterm Newborns: Value of Multiple Screening Samples and of a Detailed Clinical Characterization.

Thyroid : official journal of the American Thyroid Association
2025

Functional Noncoding Variants in a TTTG Microsatellite on Chromosome 15q26.1 Are a Common Genetic Etiology of Congenital Hypothyroidism with Thyroid Gland in situ.

Hormone research in paediatrics
2025

Transient vs Permanent Congenital Hypothyroidism: Does Thyroid Volume Tell the Tale?

Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
2025

Permanent Congenital Hypothyroidism due to Rare Thyroglobulin Gene Variant (p.Cys1476Arg): A Delayed Diagnosis of Thyroid Dyshormonogenesis.

Case reports in medicine
2025

Controlled fabrication of novel magneto-fluorescent encoded microspheres with host-guest structures for simultaneous detection of thyroxine and thyroid stimulating hormone.

Analytical methods : advancing methods and applications
2025

Variable expressivity of a transmitted pathogenic KAT6B variant.

European journal of medical genetics
2025

Genomic Characterization of Papillary Thyroid Cancer Reveals Germline Mutations Associated With Congenital Hypothyroidism.

JCO global oncology
2025

Functional Thyroid Organoids-Powerful Stem Cell-Derived Models in Basic and Translational Research.

Biomolecules
2025

Congenital Hypothyroidism: Usefulness of Technetium and Ultrasonogram of Thyroid.

Indian journal of endocrinology and metabolism
2025

Screening Blind Spot: Missing Preterm Infants in the Detection of Congenital Hypothyroidism.

International journal of neonatal screening
2025

Screening for congenital hypothyroidism by using cord blood thyroid stimulating hormone (CBTSH) levels in neonates.

Journal of family medicine and primary care
2025

Investigating TSHR gene variants in consanguineous families: novel insights into variable expression in familial congenital hypothyroidism.

Frontiers in endocrinology
2025

An Integrated Solution for Application of Next-Generation Sequencing in Newborn Screening.

Clinical laboratory
2025

From deficiency to excess: the impact of iodine excess on reproductive health.

Frontiers in endocrinology
2025

Genetic Etiology of Permanent Congenital Hypothyroidism in Korean Patients: A Whole-Exome Sequencing Study.

International journal of molecular sciences
2025

Thyroglobulin (TG) gene variants in cases with congenital goiter.

Endocrine research
2025

Vaccine hesitancy among parents of children with chronic diseases of different pathophysiology: a cross-sectional study in Sivas, Türkiye.

BMC public health
2025

Author Correction: Age of menarche and final height in patients with permanent congenital hypothyroidism.

Annals of pediatric endocrinology &amp; metabolism
2025

Early-Onset Inherited Metabolic Diseases: When Clinical Symptoms Precede Newborn Screening-Insights from Emilia-Romagna (Italy).

Children (Basel, Switzerland)
2025

Perinatal thyroid hormone deficiency leads to oxidative stress-induced neuronal damage and activation of astrocytes in rat hippocampus: Neuroprotective effect of exercise.

Neuroscience
2025

Phenotypic Characterization of Seven Pediatric Patients Diagnosed With KAT6B -Related Disorders: Case Series and Review of the Literature.

American journal of medical genetics. Part A
2025

The Establishment of Expanded Newborn Screening in Rural Areas of a Developing Country: A Model from Health Regions 7 and 8 in Thailand.

International journal of neonatal screening
2025

Liquid formulations of Levothyroxine with ethanol or with parabens for treatment of congenital hypothyroidism: similar TSH and fT4 values in the first 6 months of follow up.

Endocrine
2025

Neurodevelopmental Disorders, Cognitive Function, and Quality of Life in Children with Congenital Hypothyroidism in a Portuguese Population.

Journal of clinical research in pediatric endocrinology
2025

Screening and Functional Analysis of TPO Gene Mutations in Patients With Congenital Hypothyroidism.

Clinical genetics
2025

Unrecognized constellation of multiple congenital anomalies in a newborn: a rare case report.

Annals of medicine and surgery (2012)
2025

Imaging findings of thyroid diseases in children.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2025

Adiposity rebound and body mass index in Japanese patients with congenital hypothyroidism.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2025

CONGENITAL HYPOTHYROIDISM: FROM THEORY TO PRACTICE - A CLINICAL CASE.

Georgian medical news
2025

Simplifying Strategies to Enable Universal, Decentralized Cord Blood TSH Screening: Lessons from a Tertiary Care Center in North India.

Indian pediatrics
2025

Pseudohypoparathyroidism: Challenges in Early Recognition and Diagnosis of a Rare Hereditary Disorder.

Cureus
2025

Prevalence of Congenital Hypothyroidism and Efficacy of Current Cord Sample Screening in the Presence of Metabolic Screening: A Retrospective Cohort Study.

Cureus
2025

Predictive factors of permanent versus transient congenital hypothyroidism: a pragmatic cohort study.

Annals of pediatric endocrinology &amp; metabolism
2025

Genetics of primary congenital hypothyroidism: three decades of discoveries and persisting etiological challenges.

European thyroid journal
2025

Metabolite biomarkers of screening neonatal congenital hypothyroidism based on dried blood spot metabolomics.

Analytical and bioanalytical chemistry
2025

Congenital Hypothyroidism, Withdrawal of Thyroxin Supplementation, and Heart Failure in Dilated Cardiomyopathy: Case Report and Review of Literature.

Annals of African medicine
2025

Genetic and functional analysis of TUBB1 variants in congenital hypothyroidism.

Endokrynologia Polska
2024

Medication Adherence of Children with Congenital Hypothyroidism in Iran: A National Cross-Sectional Study.

International journal of endocrinology and metabolism
2025

Establishing a Newborn Screening Programme: A Success Story from a Low-Resource Setting.

Journal of the College of Physicians and Surgeons--Pakistan : JCPSP
2025

Neonatal screening for congenital hypothyroidism: 28-year experience in the state of Minas Gerais, Brazil.

Jornal de pediatria
2025

Newborn screening for primary congenital hypothyroidism: past, present and future.

European thyroid journal
2024

Implementing and validating newborn screening for inborn errors of metabolism in South India: a 2-year observational study at a tertiary care hospital.

BMJ public health
2024

Molecular detection of a novel mutation in the TPO gene associated with congenital hypothyroidism in a cat: Case report.

Journal of advanced veterinary and animal research
2025

Exploring the Muscle-to-Fat Ratio of Pediatric Patients with Thyroid Disorders and Its Interaction with Thyroid Function and Metabolic Syndrome Components.

Journal of clinical medicine
2025

Efficacy of levothyroxine on growth and development in children with congenital hypothyroidism: A meta-analysis.

Medicine
2025

Common and Rare DUOX Variants in Patients With Congenital Hypothyroidism: Case-control Study and Family-based Analysis.

The Journal of clinical endocrinology and metabolism
2025

Establishment of a human induced pluripotent stem cell line, KMUGMCi008-A, from a patient with A Say-Barber-Biesecker-Young-Simpson variant of Ohdo syndrome bearing heterozygous frameshift mutation in the KAT6B gene.

Stem cell research
2025

Seventh ISNS Reference Preparation for Neonatal Screening for Thyroid Stimulating Hormone, Phenylalanine, and 17α-Hydroxyprogesterone in Blood Spots.

International journal of neonatal screening
2025

Newborn Screening for Sickle Cell Disease: Results from a Pilot Study in the Portuguese Population.

International journal of neonatal screening
2025

Minimally Humanized Ezh2 Exon-18 Mouse Cell Lines Validate Preclinical CRISPR/Cas9 Approach to Treat Weaver Syndrome.

Human gene therapy
2025

Co-Occurrence of Sotos Syndrome and Mayer-Rokitansky-Küster-Hauser (MRKH) Syndrome in 2 Patients.

JCEM case reports
2024

To Study the Iodine Concentration of Mothers' Consumed Salt, Water, and Staple Food in the Neonatal Screening Program of Rural and Urban Vidarbha Region.

Journal of pharmacy &amp; bioallied sciences
2024

To Study the Thyroid Hormone Levels in Neonates of Rural and Urban Vidarbha Region.

Journal of pharmacy &amp; bioallied sciences
2025

The lived experience of migrant Syrian mothers' interaction with the neonatal screening program.

BMC public health
2025

Predictive value of technetium-99m sodium pertechnetate thyroid scintigraphy in determining the permanence of congenital hypothyroidism.

Journal of clinical imaging science
2025

Newborn screening for central congenital hypothyroidism: past, present and future.

European thyroid journal
2025

Two Novel SLC5A5 Variants (Q263L and G350D) Causing Congenital Hypothyroidism.

Thyroid : official journal of the American Thyroid Association
2025

Hypothyroid-induced hyponatraemia in a premature infant with a false-negative newborn screening for congenital hypothyroidism.

BMJ case reports
2025

Attitudes Towards the Management of Congenital Hypothyroidism in Türkiye: National Survey Study.

Journal of clinical research in pediatric endocrinology
2025

Acquired hypothyroidism, iodine status and hearing impairment in adults: A pilot study.

PloS one
2024

The Role of Neck Ultrasonography and Nuclear Imaging in the Diagnosis of Congenital Hypothyroidism.

Indian journal of endocrinology and metabolism
2025

Central congenital hypothyroidism due to TSHB gene mutation: 25-year follow-up.

BMJ case reports
2025

Pseudohypoparathyroidism type 1A presenting as short stature and congenital hypothyroidism.

Endocrinology, diabetes &amp; metabolism case reports
2025

A study on neurodevelopmental outcomes in infants with congenital hypothyroidism highlights the importance of periodic developmental assessment.

Jornal de pediatria
2025

Clinical and Demographic Factors Associated with a Delayed Thyroid-Stimulating Hormone Rise in Infants with Congenital Hypothyroidism: A Retrospective Review of a 10-Year Cohort in Indiana.

Hormone research in paediatrics
2025

Clinical Insight into Congenital Hypothyroidism Among Children.

Children (Basel, Switzerland)
2025

Newborn screening and the screening laboratory: past, present and future.

European thyroid journal
2025

Serum Neudesin Levels in Patients with Congenital Hypothyroidism.

Journal of clinical research in pediatric endocrinology
2024

Parent Reports of Developmental Service Utilization After Newborn Screening.

International journal of neonatal screening
2024

CONGENITAL HYPOTHYROIDISM SCREENING RESULTS IN THE TURKISH PROVINCE OF ADIYAMAN IN 2015-2020.

Acta endocrinologica (Bucharest, Romania : 2005)
2025

Congenital hypothyroidism and associated visual-motor and intellectual development.

Pediatric research
2025

Recovery from Atrophic Autoimmune Thyroiditis in a Child: Thyroid Stimulation-Blocking Antibody as a Prognostic Marker.

Hormone research in paediatrics
2025

Seasonal Fluctuation in the Incidence of Congenital Hypothyroidism Across Different Temperate Zones of China.

Birth defects research
2025

Unbalanced long-chain fatty acid beta-oxidation in newborns with cystic fibrosis and congenital hypothyroidism.

Molecular genetics and metabolism reports
2025

Newborn screening for congenital hypothyroidism: worldwide coverage 50 years after its start.

European thyroid journal
2025

Hypertensive disorders of pregnancy affected thyroid hormone synthesis via endoplasmic reticulum stress in preterm infant rats.

Heliyon
2025

Digenic Inheritance Mode in Congenital Hypothyroidism Due to Thyroid Dysgenesis: HYPOTYGEN Translational Cohort Study.

The Journal of clinical endocrinology and metabolism
2024

Age of menarche and final height in patients with permanent congenital hypothyroidism.

Annals of pediatric endocrinology &amp; metabolism
2024

Growth Outcomes and Final Height in Children with Acquired Hypothyroidism: A Systematic Review.

Children (Basel, Switzerland)
2024

Transient hypothyroidism in a neonate following maternal exposure to vinblastine during pregnancy: a case report and review.

Frontiers in pediatrics
2024

Functional characterization of novel compound heterozygous missense SLC5A5 gene variants causing congenital dyshormonogenic hypothyroidism.

Frontiers in endocrinology
2025

LEVOTHYROXINE-SIMETHICONE DRUG INTERACTION AND PRESENCE OF MACRO-TSH IN AN INFANT WITH CONGENITAL HYPOTHYROIDISM.

Acta endocrinologica (Bucharest, Romania : 2005)
2025

Typical endocrine disrupting chemicals in newborns with congenital hypothyroidism: Concentrations, exposure assessment, and potential risks.

Journal of hazardous materials
2024

Newborn Screening for Six Primary Conditions in a Clinical Setting in Morocco.

International journal of neonatal screening
2024

Newborn Genetic Screening Improves the Screening Efficiency for Congenital Hypothyroidism: A Prospective Multicenter Study in China.

International journal of neonatal screening
2025

Mutation spectra and genotype‑phenotype analysis of congenital hypothyroidism in a neonatal population.

Biomedical reports
2024

Diagnostic Utility of Cord Thyroid-Stimulating Hormone (TSH) in Congenital Hypothyroidism and Its Association With Perinatal Factors: A Study From a Tertiary Referral Centre in Hyderabad, India.

Cureus
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Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. A rare case report of familial glucocorticoid deficiency type 4 (GCCD4) with dilated cardiomyopathy: a 3-year follow-up study.
    Translational pediatrics· 2026· PMID 41810200mais citado
  2. Predicting Pathogenicity of TSHR Missense Variants of Uncertain Significance: An Integrative Computational Study.
    International journal of molecular sciences· 2026· PMID 41684033mais citado
  3. Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.
    Journal of tropical pediatrics· 2026· PMID 41620979mais citado
  4. Perinatal Factors Associated With Congenital Hypothyroidism in Very Low Birth Weight Infants: A Retrospective Cohort Study.
    Clinical pediatrics· 2026· PMID 40877045mais citado
  5. Congenital hypothyroidism and thyroid dysgenesis: importance of newborn bloodspot screening in Ireland.
    Irish journal of medical science· 2026· PMID 41689623mais citado
  6. [Retracted] MicroRNA-1236-3p/translationally controlled tumor protein (TPT1) axis participates in congenital hypothyroidism progression by regulating neuronal apoptosis.
    Exp Ther Med· 2026· PMID 41993152recente
  7. Congenital or Juvenile-Onset Hypothyroidism Presenting in Adulthood: A Case Series.
    Ann Afr Med· 2026· PMID 41992676recente
  8. Substrate for Thyroid Hormone Synthesis: Biochemistry, Evolution, and Physiology.
    FASEB J· 2026· PMID 41979536recente
  9. Exploring Metabolic Changes in Children with Congenital Hypothyroidism: A Serum Metabolomic Study Combined by Machine Learning.
    J Proteome Res· 2026· PMID 41973905recente
  10. Congenital hypothyroidism with preserved fifth digits in SMARCA4-related Coffin-Siris syndrome: a case report.
    AME Case Rep· 2026· PMID 41971929recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:442(Orphanet)
  2. MONDO:0018612(MONDO)
  3. Hipotiroidismo Congenito(PCDT · Ministério da Saúde)
  4. GARD:1487(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Artigo Wikipedia(Wikipedia)
  8. Q531012(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Hipotireoidismo congênito
Compêndio · Raras BR

Hipotireoidismo congênito

ORPHA:442 · MONDO:0018612
🇧🇷 Brasil SUS
Triagem
TSH neonatal em sangue seco
PNTN
Fase 1 · Nacional
Incidência BR
1:3.500
Geral
Prevalência
1-5 / 10 000
Herança
Autosomal recessive
CID-11
Ensaios
9 ativos
Medicamentos
2 registrados
Início
Antenatal, Neonatal
Prevalência
0.0 (Europe)
MedGen
UMLS
C0010308
Repurposing
1 candidato
liothyroninethyroid hormone stimulant
EuropePMC
Wikidata
Wikipedia
Papers 10a
DiscussaoAtiva

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