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Síndrome MODY
ORPHA:552CID-10 · E34.8CID-11 · 5A13.6OMIM 606391DOENÇA RARA

MODY (um tipo de diabetes que geralmente aparece em jovens ou adultos jovens) é uma forma rara e familiar de diabetes. Ele se manifesta de várias formas e é causado por diferentes alterações genéticas. Caracteriza-se por surgir em idade jovem (geralmente entre 10 e 45 anos) e por manter a produção de insulina pelo próprio corpo. Não é um diabetes autoimune (onde o sistema de defesa do corpo ataca as células do pâncreas), e geralmente não está associado à obesidade nem à resistência à insulina. Além disso, alguns tipos de MODY podem causar problemas em outras partes do corpo, além do pâncreas.

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Introdução

O que você precisa saber de cara

📋

MODY (um tipo de diabetes que geralmente aparece em jovens ou adultos jovens) é uma forma rara e familiar de diabetes. Ele se manifesta de várias formas e é causado por diferentes alterações genéticas. Caracteriza-se por surgir em idade jovem (geralmente entre 10 e 45 anos) e por manter a produção de insulina pelo próprio corpo. Não é um diabetes autoimune (onde o sistema de defesa do corpo ataca as células do pâncreas), e geralmente não está associado à obesidade nem à resistência à insulina. Além disso, alguns tipos de MODY podem causar problemas em outras partes do corpo, além do pâncreas.

Publicações científicas
1.983 artigos
Último publicado: 2026 Apr 16

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
Adolescent
+ adult, childhood
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: E34.8
Você se identifica com essa condição?
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Entender a doença

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Sinais e sintomas

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

Partes do corpo afetadas

🫘
Rins
20 sintomas
📏
Crescimento
15 sintomas
🫃
Digestivo
11 sintomas
🦴
Ossos e articulações
5 sintomas
🧠
Neurológico
3 sintomas
👂
Ouvidos
1 sintomas

+ 40 sintomas em outras categorias

Características mais comuns

55%prev.
Hiperglicemia
Frequente (79-30%)
55%prev.
Concentração anormal de insulina circulante
Frequente (79-30%)
55%prev.
Hipoinsulinemia
Frequente (79-30%)
55%prev.
Tolerância anormal à glicose oral
Frequente (79-30%)
55%prev.
Glicosúria
Frequente (79-30%)
55%prev.
Nível anormal de peptídeo C
Frequente (79-30%)
98sintomas
Frequente (8)
Ocasional (9)
Muito raro (10)
Sem dados (71)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 98 características clínicas mais associadas, ordenadas por frequência.

HiperglicemiaHyperglycemia
Frequente (79-30%)55%
Concentração anormal de insulina circulanteAbnormal circulating insulin concentration
Frequente (79-30%)55%
HipoinsulinemiaHypoinsulinemia
Frequente (79-30%)55%
Tolerância anormal à glicose oralAbnormal oral glucose tolerance
Frequente (79-30%)55%
GlicosúriaGlycosuria
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico1.983PubMed
Últimos 10 anos200publicações
Pico2025122 papers
Linha do tempo
2026Hoje · 2026🧪 1976Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

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

14 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant, Not applicable.

PAX4Paired box protein Pax-4Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Plays an important role in the differentiation and development of pancreatic islet beta cells. Transcriptional repressor that binds to a common element in the glucagon, insulin and somatostatin promoters. Competes with PAX6 for this same promoter binding site. Isoform 2 appears to be a dominant negative form antagonizing PAX4 transcriptional activity

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Regulation of gene expression in endocrine-committed (NEUROG3+) progenitor cells
MECANISMO DE DOENÇA

Type 2 diabetes mellitus

A multifactorial disorder of glucose homeostasis caused by a lack of sensitivity to insulin. Affected individuals usually have an obese body habitus and manifestations of a metabolic syndrome characterized by diabetes, insulin resistance, hypertension and hypertriglyceridemia. The disease results in long-term complications that affect the eyes, kidneys, nerves, and blood vessels.

EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
1.3 TPM
Intestino delgado
0.3 TPM
Cólon transverso
0.2 TPM
Pâncreas
0.0 TPM
Linfócitos
0.0 TPM
OUTRAS DOENÇAS (4)
maturity-onset diabetes of the young type 9type 2 diabetes mellitusmaturity-onset diabetes of the youngdiabetes mellitus, ketosis-prone
HGNC:8618UniProt:O43316
HNF1BHepatocyte nuclear factor 1-betaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor that binds to the inverted palindrome 5'-GTTAATNATTAAC-3' (PubMed:17924661, PubMed:7900999). Binds to the FPC element in the cAMP regulatory unit of the PLAU gene (By similarity). Transcriptional activity is increased by coactivator PCBD1 (PubMed:24204001)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (6)
Nephron developmentRegulation of gene expression in early pancreatic precursor cellsRegulation of gene expression in late stage (branching morphogenesis) pancreatic bud precursor cellsDevelopmental Lineage of Multipotent Pancreatic Progenitor CellsDevelopmental Lineage of Pancreatic Ductal Cells
MECANISMO DE DOENÇA

Renal cysts and diabetes syndrome

An autosomal dominant disorder comprising non-diabetic renal disease resulting from abnormal renal development, and diabetes, which in some cases occurs earlier than age 25 years and is thus consistent with a diagnosis of maturity-onset diabetes of the young (MODY5). The renal disease is highly variable and includes renal cysts, glomerular tufts, aberrant nephrogenesis, primitive tubules, irregular collecting systems, oligomeganephronia, enlarged renal pelves, abnormal calyces, small kidney, single kidney, horseshoe kidney, and hyperuricemic nephropathy. Affected individuals may also have abnormalities of the genital tract.

EXPRESSÃO TECIDUAL(Tecido-específico)
Rim - Medula
90.4 TPM
Rim - Córtex
53.5 TPM
Linfócitos
43.7 TPM
Pâncreas
23.0 TPM
Cólon transverso
14.9 TPM
OUTRAS DOENÇAS (11)
type 2 diabetes mellitusrenal cysts and diabetes syndromechromosome 17q12 deletion syndromerenal dysplasia, unilateral
HGNC:11630UniProt:P35680
CELBile salt-activated lipaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalyzes the hydrolysis of a wide range of substrates including cholesteryl esters, phospholipids, lysophospholipids, di- and tri-acylglycerols, and fatty acid esters of hydroxy fatty acids (FAHFAs) (PubMed:10220579, PubMed:27509211, PubMed:27650499, PubMed:8471055). Preferentially hydrolyzes FAHFAs with the ester bond further away from the carboxylate. Unsaturated FAHFAs are hydrolyzed more quickly than saturated FAHFAs (By similarity). Has an essential role in the complete digestion of dietar

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Developmental Lineage of Pancreatic Acinar Cells
MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 8 with exocrine dysfunction

An autosomal dominant form of diabetes characterized by a primary defect in insulin secretion, exocrine pancreatic dysfunction, altered pancreatic morphology, recurrent abdominal pain, and fecal elastase deficiency. Disease onset is at less than 25 years of age.

OUTRAS DOENÇAS (2)
maturity-onset diabetes of the young type 8maturity-onset diabetes of the young
HGNC:1848UniProt:P19835
GCKMitogen-activated protein kinase kinase kinase kinase 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Serine/threonine-protein kinase which acts as an essential component of the MAP kinase signal transduction pathway. Acts as a MAPK kinase kinase kinase (MAP4K) and is an upstream activator of the stress-activated protein kinase/c-Jun N-terminal kinase (SAP/JNK) signaling pathway and to a lesser extent of the p38 MAPKs signaling pathway. Required for the efficient activation of JNKs by TRAF6-dependent stimuli, including pathogen-associated molecular patterns (PAMPs) such as polyinosine-polycytidi

LOCALIZAÇÃO

CytoplasmBasolateral cell membraneGolgi apparatus membrane

VIAS BIOLÓGICAS (5)
GlycolysisRegulation of gene expression in beta cellsRegulation of Glucokinase by Glucokinase Regulatory ProteinDefective TPR may confer susceptibility towards thyroid papillary carcinoma (TPC)FOXO-mediated transcription of oxidative stress, metabolic and neuronal genes
EXPRESSÃO TECIDUAL(Tecido-específico)
Pituitária
34.5 TPM
Cérebro - Hemisfério cerebelar
7.2 TPM
Cerebelo
7.1 TPM
Hipotálamo
6.5 TPM
Coração - Átrio
3.1 TPM
OUTRAS DOENÇAS (7)
type 2 diabetes mellitusmaturity-onset diabetes of the young type 2permanent neonatal diabetes mellitus 1monogenic diabetes
HGNC:4195UniProt:Q12851
PDX1Pancreas/duodenum homeobox protein 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Activates insulin, somatostatin, glucokinase, islet amyloid polypeptide and glucose transporter type 2 gene transcription. Particularly involved in glucose-dependent regulation of insulin gene transcription. As part of a PDX1:PBX1b:MEIS2b complex in pancreatic acinar cells is involved in the transcriptional activation of the ELA1 enhancer; the complex binds to the enhancer B element and cooperates with the transcription factor 1 complex (PTF1) bound to the enhancer A element. Binds preferentiall

LOCALIZAÇÃO

NucleusCytoplasm, cytosol

VIAS BIOLÓGICAS (5)
Regulation of gene expression in beta cellsRegulation of gene expression in early pancreatic precursor cellsDevelopmental Lineage of Multipotent Pancreatic Progenitor CellsDevelopmental Lineage of Pancreatic Ductal CellsDevelopmental Lineage of Pancreatic Acinar Cells
MECANISMO DE DOENÇA

Pancreatic agenesis 1

A disease characterized by isolated hypoplasia or agenesis of the pancreas, pancreatic beta-cell failure resulting in neonatal insulin-dependent diabetes mellitus, and exocrine pancreatic insufficiency.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pâncreas
9.2 TPM
Linfócitos
0.5 TPM
Testículo
0.2 TPM
Fígado
0.2 TPM
Estômago
0.1 TPM
OUTRAS DOENÇAS (6)
maturity-onset diabetes of the young type 4pancreatic agenesis 1pancreatic agenesismaturity-onset diabetes of the young
HGNC:6107UniProt:P52945
ABCC8ATP-binding cassette sub-family C member 8Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Regulator subunit of pancreatic ATP-sensitive potassium channel (KATP), playing a major role in the regulation of insulin release. In pancreatic cells, it forms KATP channels with KCNJ11; KCNJ11 forms the channel pore while ABCC8 is required for activation and regulation

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (2)
Regulation of insulin secretionATP sensitive Potassium channels
MECANISMO DE DOENÇA

Leucine-induced hypoglycemia

Rare cause of hypoglycemia and is described as a condition in which symptomatic hypoglycemia is provoked by high protein feedings. Hypoglycemia is also elicited by administration of oral or intravenous infusions of a single amino acid, leucine.

OUTRAS DOENÇAS (12)
hyperinsulinemic hypoglycemia, familial, 1diabetes mellitus, transient neonatal, 2diabetes mellitus, permanent neonatal 3hypoglycemia, leucine-induced
HGNC:59UniProt:Q09428
KCNJ11ATP-sensitive inward rectifier potassium channel 11Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Inward rectifier potassium channel that forms the pore of ATP-sensitive potassium channels (KATP), regulating potassium permeability as a function of cytoplasmic ATP and ADP concentrations in many different cells (PubMed:29286281, PubMed:34815345). Inward rectifier potassium channels are characterized by a greater tendency to allow potassium to flow into the cell rather than out of it. Their voltage dependence is regulated by the concentration of extracellular potassium; as external potassium is

LOCALIZAÇÃO

Membrane

VIAS BIOLÓGICAS (6)
Ion homeostasisABC-family proteins mediated transportDefective ABCC9 causes CMD10, ATFB12 and Cantu syndromeDefective ABCC8 can cause hypo- and hyper-glycemiasRegulation of insulin secretion
MECANISMO DE DOENÇA

Hyperinsulinemic hypoglycemia, familial, 2

A form of hyperinsulinemic hypoglycemia, a clinically and genetically heterogeneous disorder characterized by inappropriate insulin secretion from the pancreatic beta-cells in the presence of low blood glucose levels. HHF2 is a common cause of persistent hypoglycemia in infancy. Unless early and aggressive intervention is undertaken, brain damage from recurrent episodes of hypoglycemia may occur. HHF2 inheritance can be autosomal dominant or autosomal recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Músculo esquelético
87.1 TPM
Cerebelo
37.4 TPM
Cérebro - Hemisfério cerebelar
36.8 TPM
Córtex cerebral
14.0 TPM
Brain Frontal Cortex BA9
13.9 TPM
OUTRAS DOENÇAS (12)
maturity-onset diabetes of the young type 13diabetes mellitus, permanent neonatal 2hyperinsulinemic hypoglycemia, familial, 2diabetes mellitus, transient neonatal, 3
HGNC:6257UniProt:Q14654
BLKTyrosine-protein kinase BlkDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Non-receptor tyrosine kinase involved in B-lymphocyte development, differentiation and signaling (By similarity). B-cell receptor (BCR) signaling requires a tight regulation of several protein tyrosine kinases and phosphatases, and associated coreceptors (By similarity). Binding of antigen to the B-cell antigen receptor (BCR) triggers signaling that ultimately leads to B-cell activation (By similarity). Signaling through BLK plays an important role in transmitting signals through surface immunog

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (1)
RUNX1 regulates transcription of genes involved in BCR signaling
MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 11

A form of diabetes that is characterized by an autosomal dominant mode of inheritance, onset in childhood or early adulthood (usually before 25 years of age), a primary defect in insulin secretion and frequent insulin-independence at the beginning of the disease.

OUTRAS DOENÇAS (3)
maturity-onset diabetes of the young type 11systemic lupus erythematosusmaturity-onset diabetes of the young
HGNC:1057UniProt:P51451
HNF4AHepatocyte nuclear factor 4-alphaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcriptional regulator which controls the expression of hepatic genes during the transition of endodermal cells to hepatic progenitor cells, facilitating the recruitment of RNA pol II to the promoters of target genes (PubMed:30597922). Activates the transcription of CYP2C38 (By similarity). Represses the CLOCK-BMAL1 transcriptional activity and is essential for circadian rhythm maintenance and period regulation in the liver and colon cells (PubMed:30530698)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (2)
Nuclear Receptor transcription pathwayNephron development
MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 1

A form of diabetes that is characterized by an autosomal dominant mode of inheritance, onset in childhood or early adulthood (usually before 25 years of age), a primary defect in insulin secretion and frequent insulin-independence at the beginning of the disease.

EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
55.4 TPM
Cólon transverso
33.0 TPM
Intestino delgado
30.7 TPM
Rim - Córtex
11.4 TPM
Pâncreas
5.6 TPM
OUTRAS DOENÇAS (7)
maturity-onset diabetes of the young type 1Fanconi renotubular syndrome 4 with maturity-onset diabetes of the youngmonogenic diabetesatypical Fanconi syndrome-neonatal hyperinsulinism syndrome
HGNC:5024UniProt:P41235
KLF11Krueppel-like factor 11Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Transcription factor (PubMed:10207080, PubMed:9748269). Activates the epsilon- and gamma-globin gene promoters and, to a much lower degree, the beta-globin gene and represses promoters containing SP1-like binding inhibiting cell growth (PubMed:10207080, PubMed:16131492, PubMed:9748269). Represses transcription of SMAD7 which enhances TGF-beta signaling (By similarity). Induces apoptosis (By similarity)

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 7

A form of diabetes that is characterized by an autosomal dominant mode of inheritance, onset in childhood or early adulthood (usually before 25 years of age), a primary defect in insulin secretion and frequent insulin-independence at the beginning of the disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
43.6 TPM
Tecido adiposo
42.3 TPM
Adipose Visceral Omentum
38.4 TPM
Skin Not Sun Exposed Suprapubic
35.0 TPM
Mama
34.6 TPM
INTERAÇÕES PROTEICAS (5)
OUTRAS DOENÇAS (2)
maturity-onset diabetes of the young type 7maturity-onset diabetes of the young
HGNC:11811UniProt:O14901
INSInsulinDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Insulin decreases blood glucose concentration. It increases cell permeability to monosaccharides, amino acids and fatty acids. It accelerates glycolysis, the pentose phosphate cycle, and glycogen synthesis in liver

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
Insulin receptor recyclingSignaling by Insulin receptor
MECANISMO DE DOENÇA

Hyperproinsulinemia

An autosomal dominant condition characterized by elevated levels of serum proinsulin-like material.

EXPRESSÃO TECIDUAL(Tecido-específico)
Pâncreas
2325.3 TPM
Glândula adrenal
0.5 TPM
Cervix Ectocervix
0.5 TPM
Substância negra
0.4 TPM
Baço
0.3 TPM
OUTRAS DOENÇAS (6)
maturity-onset diabetes of the young type 10diabetes mellitus, permanent neonatal 4hyperproinsulinemiatype 1 diabetes mellitus 2
HGNC:6081UniProt:P01308
APPL1DCC-interacting protein 13-alphaDisease-causing germline mutation(s) (loss of function) inRestrito
FUNÇÃO

Multifunctional adapter protein that binds to various membrane receptors, nuclear factors and signaling proteins to regulate many processes, such as cell proliferation, immune response, endosomal trafficking and cell metabolism (PubMed:10490823, PubMed:15016378, PubMed:19661063, PubMed:26073777, PubMed:26583432). Regulates signaling pathway leading to cell proliferation through interaction with RAB5A and subunits of the NuRD/MeCP1 complex (PubMed:15016378). Functions as a positive regulator of i

LOCALIZAÇÃO

Early endosome membraneNucleusCytoplasmEndosomeCell projection, ruffleCytoplasmic vesicle, phagosome

VIAS BIOLÓGICAS (1)
Caspase activation via Dependence Receptors in the absence of ligand
MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 14

A form of diabetes that is characterized by an autosomal dominant mode of inheritance, onset in childhood or early adulthood (usually before 25 years of age), a primary defect in insulin secretion and frequent insulin-independence at the beginning of the disease.

OUTRAS DOENÇAS (2)
maturity-onset diabetes of the youngmaturity-onset diabetes of the young type 14
HGNC:24035UniProt:Q9UKG1
HNF1AHepatocyte nuclear factor 1-alphaDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcriptional activator that regulates the tissue specific expression of multiple genes, especially in pancreatic islet cells and in liver (By similarity). Binds to the inverted palindrome 5'-GTTAATNATTAAC-3' (PubMed:10966642, PubMed:12453420). Activates the transcription of CYP1A2, CYP2E1 and CYP3A11 (By similarity) (Microbial infection) Plays a crucial role for hepatitis B virus gene transcription and DNA replication. Mechanistically, synergistically cooperates with NR5A2 to up-regulate the

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Regulation of gene expression in beta cells
MECANISMO DE DOENÇA

Hepatic adenomas familial

Rare benign liver tumors of presumable epithelial origin that develop in an otherwise normal liver. Hepatic adenomas may be single or multiple. They consist of sheets of well-differentiated hepatocytes that contain fat and glycogen and can produce bile. Bile ducts or portal areas are absent. Kupffer cells, if present, are reduced in number and are non-functional. Conditions associated with adenomas are insulin-dependent diabetes mellitus and glycogen storage diseases (types 1 and 3).

EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
8.7 TPM
Intestino delgado
6.3 TPM
Rim - Córtex
5.3 TPM
Cólon transverso
4.3 TPM
Estômago
4.1 TPM
OUTRAS DOENÇAS (11)
maturity-onset diabetes of the young type 3nonpapillary renal cell carcinomahepatic adenomas, familialtype 1 diabetes mellitus 20
HGNC:11621UniProt:P20823
NEUROD1Neurogenic differentiation factor 1Disease-causing germline mutation(s) inModerado
FUNÇÃO

Acts as a transcriptional activator: mediates transcriptional activation by binding to E box-containing promoter consensus core sequences 5'-CANNTG-3'. Associates with the p300/CBP transcription coactivator complex to stimulate transcription of the secretin gene as well as the gene encoding the cyclin-dependent kinase inhibitor CDKN1A. Contributes to the regulation of several cell differentiation pathways, like those that promote the formation of early retinal ganglion cells, inner ear sensory n

LOCALIZAÇÃO

CytoplasmNucleus

VIAS BIOLÓGICAS (2)
Regulation of gene expression in beta cellsRegulation of gene expression in endocrine-committed (NEUROG3+) progenitor cells
MECANISMO DE DOENÇA

Maturity-onset diabetes of the young 6

A form of diabetes that is characterized by an autosomal dominant mode of inheritance, onset in childhood or early adulthood (usually before 25 years of age), a primary defect in insulin secretion and frequent insulin-independence at the beginning of the disease.

EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
306.4 TPM
Cerebelo
215.4 TPM
Hipocampo
4.2 TPM
Brain Frontal Cortex BA9
3.6 TPM
Córtex cerebral
3.4 TPM
OUTRAS DOENÇAS (3)
maturity-onset diabetes of the young type 6maturity-onset diabetes of the youngtype 2 diabetes mellitus
HGNC:7762UniProt:Q13562

Variantes genéticas (ClinVar)

660 variantes patogênicas registradas no ClinVar.

🧬 PAX4: GRCh37/hg19 7q31.32-36.1(chr7:122190535-149944340)x1 ()
🧬 PAX4: GRCh37/hg19 7q31.31-33(chr7:120582003-137699953)x1 ()
🧬 PAX4: NM_001366110.1(PAX4):c.761T>C (p.Ile254Thr) ()
🧬 PAX4: GRCh37/hg19 7q31.33-32.2(chr7:127076892-129405807)x1 ()
🧬 PAX4: NM_001366110.1(PAX4):c.14-2A>G ()
Ver todas no ClinVar

Vias biológicas (Reactome)

33 vias biológicas associadas aos genes desta condição.

Regulation of gene expression in endocrine-committed (NEUROG3+) progenitor cells Regulation of gene expression in late stage (branching morphogenesis) pancreatic bud precursor cells Regulation of gene expression in early pancreatic precursor cells Nephron development Developmental Lineage of Pancreatic Acinar Cells Developmental Lineage of Pancreatic Ductal Cells Developmental Lineage of Multipotent Pancreatic Progenitor Cells Digestion of dietary lipid Defective GCK causes maturity-onset diabetes of the young 2 (MODY2) Defective HK1 causes hexokinase deficiency (HK deficiency) FOXO-mediated transcription of oxidative stress, metabolic and neuronal genes Regulation of gene expression in beta cells ATP sensitive Potassium channels Regulation of insulin secretion Defective ABCC8 can cause hypo- and hyper-glycemias ABC-family proteins mediated transport Ion homeostasis Defective ABCC9 causes CMD10, ATFB12 and Cantu syndrome RUNX1 regulates transcription of genes involved in BCR signaling Antigen activates B Cell Receptor (BCR) leading to generation of second messengers Nuclear Receptor transcription pathway Insulin processing Synthesis, secretion, and deacylation of Ghrelin COPI-mediated anterograde transport PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling IRS activation Signal attenuation Insulin receptor signalling cascade Signaling by Insulin receptor Insulin receptor recycling NPAS4 regulates expression of target genes Amyloid fiber formation Caspase activation via Dependence Receptors in the absence of ligand

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

🇧🇷 Atendimento SUS — Síndrome MODY

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Publicações mais relevantes

Timeline de publicações
1.087 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 1.087

#1

Development of a Calculator for HNF1A- and HNF4A-MODY in Asian Indians.

Journal of diabetes science and technology2026 Mar 23

We aimed to develop a calculator to determine the probability of having HNF1A-MODY (hepatocyte nuclear factor 1 alpha-maturity-onset diabetes of the young) or HNF4A (hepatocyte nuclear factor 4 alpha)-MODY (the commonest forms of MODY) in Asian Indians using clinical and biochemical criteria. We extracted data on individuals with young-onset diabetes aged <30 years (n = 29 191) from electronic records. Genetically confirmed HNF1A- and HNF4A-MODY (n = 55) were selected along with 1000 individuals each of type 1 diabetes (T1D) and type 2 diabetes (T2D). These data sets were used to develop a classification model using logistic regression. The model's performance was evaluated using receiver operating characteristic (ROC) curves in an internal data set and validated in an external data set. Eight predictive models were constructed, beginning with a basic model that included variables, such as age at diagnosis, body mass index (BMI), parental history, and glycated hemoglobin (HbA1c) (models 1 and 5). High-density lipoprotein (HDL) cholesterol was added in models 2 and 6, stimulated C-peptide in models 3 and 7, and all predictors were combined in models 4 and 8. Models 1 to 4, designed to distinguish MODY from T1D, achieved an ROC-area under the curve (AUC) value ranging from 0.884 to 0.957, while models 5 to 8, aimed at differentiating MODY from T2D, achieved an ROC-AUC value ranging from 0.914 to 0.936. All models demonstrated excellent performance in internal validation, with high five-fold cross-validation c-statistics. An online calculator using these models estimates MODY probability that is accessible at https://mdrf-t1d-calculator.shinyapps.io/MODY/. We developed an ethnicity-specific calculator to help identify individuals with possible HNF1A-MODY or HNF4A-MODY in Asian Indians. This user-friendly, web-based tool would be helpful to select candidates for genetic testing in this population.

#2

Discovery and preclinical activity of the menin-KMT2A inhibitor ziftomenib in acute leukemia models.

Blood2026 Mar 18

The protein-protein interaction between menin and KMT2A (histone lysine methyltransferase 2A) plays a critical role in acute leukemia with KMT2A rearrangements, nucleophosmin 1 (NPM1) mutations and nucleoporin 98 rearrangements, and represents an emerging opportunity for therapeutic intervention. Here, we report development and comprehensive evaluation of the activity of ziftomenib as an orally bioavailable, highly potent and selective small molecule inhibitor of the menin-KMT2A interaction. In leukemia cells and primary patient samples with the menin-KMT2A dependency, ziftomenib profoundly inhibited proliferation, reduced clonogenic potential and induced differentiation, which was associated with strong downregulation of the menin-KMT2A target genes, including MEIS1, HOXA9 and HOXB2. In xenografts and patient-derived xenograft models of KMT2A-rearranged leukemia, ziftomenib induced leukemia regression or reduced leukemia burden, accompanied by a pronounced reduction of the menin-KMT2A target genes. We next assessed ziftomenib against four MEN1 (gene encoding menin) mutants (T349M, M327I, G331R, G331D) associated with clinical resistance to another menin inhibitor revumenib. Ziftomenib retained anti-leukemic activity against T349M mutant cells and demonstrated low-nanomolar potency (GI50≤25 nM) against G331R cells, despite several-fold reduced potency relative to MEN1 wild-type cells, whereas M327I and G331D mutants were resistant. The crystal structures of ziftomenib in complex with menin wild-type, T349M or G331R mutants revealed a similar binding mode of ziftomenib to these menin variants, rationalizing potent inhibitory activity towards these mutants. Ziftomenib has recently received FDA approval for adult patients with NPM1-mutated acute myeloid leukemia and continues to be evaluated clinically in leukemias with NPM1 or KMT2A alterations, both as monotherapy and in combinations.

#3

Clinical Characterization of Atypical Diabetes: Insights from the GENEPEDIAB Study into the Spectrum Between Type 1 and Monogenic Diabetes.

Cells2026 Mar 07

Type 1 diabetes (T1D) shares clinical characteristics with other forms of diabetes, particularly monogenic diabetes such as maturity-onset diabetes of the young (MODY). Differential diagnosis is complicated by the existence of intermediate phenotypes. We aimed to delineate the phenotypic continuum between T1D and monogenic diabetes. The multicentric GENEPEDIAB study included patients aged 6 months to 18 years diagnosed with diabetes and treated for either T1D or monogenic diabetes. Analyses comprised glycemic variability, continuous glucose monitoring metrics, application of the DIAMODIA criteria, and genetic investigations. A gradient was observed across T1D, atypical diabetes (Adia), and MODY cohorts for several glycemic parameters. T1D patients exhibited values furthest from treatment targets, whereas MODY patients showed better glycemic control. Stratification of the Adia cohort according to the number of positive DIAMODIA criteria further supported this trend, as demonstrated by glycemic measures and multiple correspondence analysis. Genetic analyses did not identify a uniform causative variant in the Adia cohort; however, several rare variants, including variants of uncertain significance and likely pathogenic variants in diabetes-related genes, were detected. These findings showed, in our specific cohort of pediatric patients, the existence of a phenotypic gradient between T1D and monogenic diabetes, with atypical diabetes occupying an intermediate position, including when stratified by DIAMODIA criteria.

#4

Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High-Risk Neuroblastoma: A Multi-Institutional Retrospective Study.

Pediatric blood &amp; cancer2026 Mar 13

Chemoimmunotherapy with irinotecan, temozolomide, and dinutuximab (I/T/DIN) has emerged as first-line therapy for relapsed/refractory (r/r) high-risk neuroblastoma (HRNB) in North America. Topotecan and cyclophosphamide (T/C) are often used in combination with dinutuximab in the setting of lack of response, progression, or incomplete response following I/T/DIN, but efficacy and tolerability are unknown. Eligible patients received one or more cycles of T/C/DIN for r/r HRNB. Response was assessed using the 2017 International Neuroblastoma Response Criteria. Fifty-four patients with r/r HRNB were included. Patients received a median of four cycles of T/C/DIN (range = 1-16); 48 (89%) had previously received I/T/DIN. Twenty-three patients (42.6%) had an objective response (complete/partial/minor; OR) to T/C/DIN, 21 (38.9%) had stable disease, and 10 (18.5%) had progressive disease as best response. Among patients who previously received I/T/DIN, objective response rate (ORR) to T/C/DIN was 54% in those with prior OR to I/T/DIN and 23% in those without prior OR (adjusted odds ratio 4.40, 95% confidence interval [CI]: 1.09-17.75). Patients who had experienced on-therapy progression with I/T/DIN had a similar ORR to T/C/DIN compared to those who never progressed (38% vs. 41%, p = 1.0). Twenty-one patients (39%) required T/C/DIN dose modification-11 (52%) due to hematologic toxicity. Seven patients (13%) discontinued T/C/DIN due to toxicity. T/C/DIN is an effective regimen for patients with r/r HRNB, producing ORRs similar to prior reports in chemoimmunotherapy-naïve patients treated with I/T/DIN. Prior chemoimmunotherapy response may predict response to T/C/DIN, but even patients who progress while receiving I/T/DIN may benefit from the T/C/DIN combination.

#5

A customized CNN model for signature authentication-Forensic implications.

Medicine, science, and the law2026 Feb 13

Signature authentication refers to the verification whether the signature is legitimate or forged and is executed by the person who is claiming to have signed it. The main objective of the present research was to customize a deep learning-based convolutional neural network (CNN) model trained on a primary dataset for signature authentication. The model was trained, validated, and tested on the dataset of the 1400 signature images (700 genuine and 700 forged) primarily obtained from the study participants. The signature samples were equally divided into a train (1000 samples comprising 500 forged and 500 genuine signatures) and a test dataset (400 samples comprising 200 forged and 200 genuine signatures) as per the requirements of the model's training and testing procedure. The model's architecture was optimized with different hyperparameters to achieve a higher accuracy rate. The results show that the proposed model attains the training, validation and testing accuracy of 97.32%, 97.92%, and 84.5% respectively. Furthermore, other evaluation matrices were also computed, including precision, recall (sensitivity), F1-score, and specificity with the values of 85%, 84%, 84%, and 90%, respectively. The accuracy matrices provide better performance over the other existing methods. This customized CNN architecture can be trained on larger datasets and directly deployed in the field of forensic science for signature examination. The study has wide-ranging applications in the banking sector, forensic document examination, courtrooms, and beyond.

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📚 EuropePMC752 artigos no totalmostrando 194

2026

The effect of vericiguat on sudden cardiac death: insights from the VICTOR trial.

European journal of heart failure
2026

Development of a Calculator for HNF1A- and HNF4A-MODY in Asian Indians.

Journal of diabetes science and technology
2026

Age- and diet-dependent progression of retinal microvascular injury in GCK-MODY under metabolic stress.

Frontiers in endocrinology
2026

Nanotechnology-Based Strategies for Glioblastoma: Diagnostic and Therapeutic Advances.

Current neuropharmacology
2026

Progress of HIV care cascade and transition dynamics along the continuum of care among persons living with HIV in Ifakara, Tanzania: a prospective study.

BMC infectious diseases
2026

Discovery and preclinical activity of the menin-KMT2A inhibitor ziftomenib in acute leukemia models.

Blood
2026

Polygenic background contributes to GCK-MODY clinical presentation and glycaemic variability.

Diabetologia
2026

Diverse and rare candidate MODY gene variants were identified in one-fifth of a Bangladeshi cohort with nonobese, nonautoimmune youth-onset diabetes.

Scientific reports
2026

Clinical Predictors of Nose/throat Bacteriome and Fungal Colonization in Skilled Nursing Facility Residents.

The Journal of infectious diseases
2026

Clinical Characterization of Atypical Diabetes: Insights from the GENEPEDIAB Study into the Spectrum Between Type 1 and Monogenic Diabetes.

Cells
2026

Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High-Risk Neuroblastoma: A Multi-Institutional Retrospective Study.

Pediatric blood &amp; cancer
2026

Survival after salvage pulmonary resection for non-small cell lung cancer: a national cancer database analysis.

Journal of thoracic disease
2025

Lipid-dependent uncoupling of agonist binding and channel gating in the nicotinic acetylcholine receptor.

Biophysical reviews
2026

Case Report: Identification of a HNF1A exons 1-10 heterozygous deletion in a Chinese MODY family.

Frontiers in endocrinology
2026

Development, optimization, and in vitro assessment of abuse-deterrent formulation.

Journal of opioid management
2026

Systematic analysis of loss-of-function variants across MODY genes demonstrates gene-specific effects and expands the spectrum of INS variants causing MODY.

Diabetologia
2026

Genotype-phenotype correlations of fasting C-peptide and lipids in HNF1A-MODY: a single-center series and multi-center cross-sectional analysis in Chinese population.

Frontiers in endocrinology
2026

Deciphering Silence: Functional Studies of GCK Synonymous and Nonsense Variants and Their Importance in Understanding Diabetes.

Genes
2026

Review Article: Overview of Clinical Genetics of Diabetes Mellitus.

Genes
2026

Integrated transcriptome and proteome analyses unveil cytoskeletal alterations in an endothelial model of monogenic diabetes.

Genome medicine
2026

Experimental quantification of resistance to subsidence conferred by endplate-specific expandable implants in anterior lumbar interbody fusion.

Journal of orthopaedic surgery and research
2026

Low prevalence of GCK gene mutations in Chinese patients with gestational diabetes mellitus.

Clinics (Sao Paulo, Brazil)
2026

Protocol for automated analysis of biological images using Python code.

STAR protocols
2025

Approach to large thrombus burden in ST-elevation myocardial infarction.

Frontiers in cardiovascular medicine
2026

Applications of genetic testing in cardiovascular disease.

Current opinion in cardiology
2026

Reduced Liver Fat Content in GCK-MODY Patients Compared With Healthy Individuals Without Diabetes.

Diabetes care
2026

High prevalence of maturity-onset diabetes of the young in the Czech Republic: A 25-year nationwide registry-based study.

Diabetic medicine : a journal of the British Diabetic Association
2026

RFX6 expression is central to the development and function of the neuroendocrine compartments of the pancreas and intestine and strongly affects diabetes risk.

Diabetology international
2026

Probing the effects of protein glycosylation on transcription with induced-proximity tools.

Nature reviews. Molecular cell biology
2025

Case Report: Nephrocalcinosis from pancreatic hypoplasia in HNF1B disease: a multigenerational expression with genetic confirmation in the youngest generation.

Frontiers in medicine
2026

Identification of maturity-onset diabetes of the young through targeted next-generation sequencing in Thai patients with atypical diabetes in real-world practice.

Frontiers in endocrinology
2026

C-peptide in Precision Diabetes Care and Beyond: A Comprehensive Review.

Clinical medicine insights. Endocrinology and diabetes
2026

Endocrine Care During Transition in Emerging Adults With Cystic Fibrosis: A Single Center Retrospective Study.

Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
2026

A customized CNN model for signature authentication-Forensic implications.

Medicine, science, and the law
2026

Search for the most precise diagnosis of monogenic diabetes - the usefulness of short-read NGS method in molecular testing in Polish patients.

Acta diabetologica
2026

Progression of diabetic retinopathy during pregnancy in a woman with ABCC8-MODY.

Endocrinology, diabetes &amp; metabolism case reports
2026

HLA-guided identification of monogenic diabetes in antibody-negative type 1 diabetes: frequency and characteristics.

Diabetes &amp; metabolism
2026

Ticagrelor vs Prasugrel in Patients With Diabetes and Multivessel Coronary Artery Disease: The TUXEDO-2 Randomized Clinical Trial.

JAMA cardiology
2026

Utility of next-generation sequencing for the genetic study of MODY diabetes.

The Journal of endocrinology
2026

Reducing Potentially Inappropriate Medication Use for Older Adults in the Emergency Department-A Safer Script.

JAMA internal medicine
2026

SARS-CoV-2 infection and sleep health in Military Health System beneficiaries.

Sleep advances : a journal of the Sleep Research Society
2026

Novel variation in the CEL gene causing impaired fasting glucose in a Chinese pediatric patient: case report and literature review.

Frontiers in endocrinology
2026

Evaluation of rare NR1D2 variants in MODY-X: clinical, genetic, and in silico insights.

Journal of endocrinological investigation
2025

Antiphospholipid Syndrome in Orthopaedic Foot and Ankle Surgery: A Propensity-Matched Analysis.

Foot &amp; ankle orthopaedics
2026

RFX6 maturity-onset diabetes of the young: clinical considerations and novel use of tirzepatide.

Endocrinology, diabetes &amp; metabolism case reports
2025

Novel variants of monogenic diabetes and impact of genetic diagnosis on treatment strategies.

Frontiers in medicine
2026

Comprehensive Phenotype and Treatment Description of Mitochondrial Diabetes: Insights From a Large Cohort Study.

Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
2026

Burden of malaria among children aged 5-10 years in the Sahelian area: do we need to adapt seasonal malaria chemoprophylaxis?

BMC infectious diseases
2025

Genomic Insights into Unspecified Monogenic Forms of Diabetes and Their Associated Comorbidities: Implication for Treatment.

Current issues in molecular biology
2026

Genetic diversity and molecular mechanisms in hypertrophic cardiomyopathy: toward personalized therapy.

American journal of physiology. Heart and circulatory physiology
2026

Association Between Non-Tobacco Nicotine Dependence and Postoperative Complications After Ankle and Hindfoot Arthrodesis.

Foot &amp; ankle international
2026

Study of the ovarian function and gyneco-obstetrical profile of patients with an HNF1B abnormality.

European journal of obstetrics, gynecology, and reproductive biology
2025

Analysis of Mutation Site Structure Prediction and Family Characteristics of Maturity-Onset Diabetes of the Young (MODY) with Ketosis: Caused by HNF4α Gene Mutation.

Neuro endocrinology letters
2026

Effect of class IV LASER therapy along with Low-dye kinesio-taping technique or plantar fascia stretching on pain, pain pressure threshold, and disability in individuals with plantar fasciitis: Double-blinded randomized clinical trial.

Lasers in medical science
2026

PrEParing for Long-acting Technologies: A Multistate Analysis of PrEP Persistence and HIV and STI Lab Coverage Among Oral PrEP Initiators in St. Louis, Missouri (2014-2021).

Open forum infectious diseases
2026

Clinical outcomes in acute myocardial infarction cardiogenic shock patients supported with the Impella 5.5, high-flow, surgically implanted micro-axial flow pump.

The Journal of thoracic and cardiovascular surgery
2026

Successful Use of Recombinant Tissue Plasminogen Activator in an Extremely Low Birth Weight Premature Infant for the Resolution of Multiple Thrombi.

AJP reports
2025

Comparing expert assessments of research quality between the Global North and East Africa.

Philosophical transactions of the Royal Society of London. Series B, Biological sciences
2026

PDX1 phosphorylation at S61 by mTORC1 links nutrient signaling to β cell function and metabolic disease.

Cell reports
2026

Small molecule splicing modulators that disrupt O-GlcNAc homeostasis.

Nature communications
2026

Empagliflozin in HNF1A-MODY (MODY3)-a Randomized, Double-Blind, Placebo-Controlled, Crossover Trial.

Diabetes care
2026

Skilled Nursing Facilities and Sufficient Bed Capacity-No Room at the Inn.

JAMA internal medicine
2026

The effect of GLP-1 receptor agonists on cognition in nondiabetic patients with mild cognitive impairment or alzheimer's disease: a meta-analysis of randomized controlled trials.

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
2025

Functional Characterization of Glucokinase Variants to Aid Clinical Interpretation of Monogenic Diabetes.

International journal of molecular sciences
2025

Genetic Characterization of MODY in Iranian Families Using Multigenerational-Based Whole-Exome Sequencing Approach.

Journal of diabetes research
2026

Breakpoint-resolved balanced t(2;12)(q35;q24.31) disrupting HNF1A in multigenerational MODY-3: Diagnostic utility of long-read genome sequencing and therapeutic impact.

Metabolism open
2026

Optimizing SMILES token sequences via trie-based refinement and transition graph filtering.

Journal of cheminformatics
2025

dentification and characterization of novel PAX4 variants in patients with suspected MODY9.

BMJ open diabetes research &amp; care
2025

Concurrent hepatic adenomatosis and hemangiomatosis in a patient with HNF1A MODY.

BMJ case reports
2025

Management and outcomes in pregnant patients with monogenic diabetes due to pathogenic variants in GCK and HNF1A genes.

Frontiers in endocrinology
2025

Composite Digenic Diabetes Linked to Heterozygous Variants of <italic>GCK</italic> and <italic>NEUROD1</italic>: A Case Report.

Hormone research in paediatrics
2025

Non-type 1 and non-type 2 diabetes in a young man due to novel mutation in HNF1A.

BMJ case reports
2025

Depression and the revolving door of HIV care: using multistate approaches to understand complex longitudinal exposure/outcome relationships among a cohort of people entering HIV care in Cameroon from 2019 to 2020.

BMJ public health
2026

Duration-dependent alterations of lipid profiles and microvascular complications in GCK-MODY.

Biochimica et biophysica acta. Molecular basis of disease
2025

Gliclazide Challenge Testing as an Alternative Diagnostic Tool for Hepatocyte Nuclear Factor-1-Alpha Maturity Onset Diabetes of the Young.

European journal of case reports in internal medicine
2025

Exome Sequencing of a Type 1 Diabetes Mellitus Family Exposes Both Common and Individualized Rare Variants Contributing to Pathogenesis.

Journal of diabetes research
2025

Inflammatory arthritis in people living with HIV on anti-retroviral therapy in a high burden setting in South Africa.

HIV research &amp; clinical practice
2025

Revealing Monogenic Diabetes: Clinical and Genetic Features of Pediatric MODY Cases in Türkiye: Single Center Experience.

Pediatric diabetes
2025

Coexistence of Maturity-Onset Diabetes of the Young Type 3 (MODY 3) and Diabetic Ketoacidosis: A Report of a Rare Case.

Cureus
2025

Targeted protein O-GlcNAc reveals transcriptional functions for O-GlcNAc.

Cell chemical biology
2025

Why all MODY variants in transcription factor genes are dominantly inherited.

Frontiers in genetics
2025

Comparing auditory vs. virtual reality relaxation in reducing dental anxiety.

Frontiers in dental medicine
2025

Trends and clinical features of childhood diabetes subgroups: 28 years of single center experience.

The Turkish journal of pediatrics
2025

Pediatric Oncology Patients With Germline Pathogenic Variants in Adult-Onset Cancer Predisposition Genes.

JCO precision oncology
2026

The role of small, dense lipoproteins in type-2 diabetes and maturity-onset diabetes of the young (MODY): What's new.

Journal of diabetes and its complications
2026

The TRANSEVER registry - A prospective, open-label, multicentre, post market surveillance study of ISAR SUMMIT polymer-free Everolimus eluting stent in a real-world Indian population of patients with coronary artery disease: One-year outcomes of the first 500 enrolled patients.

International journal of cardiology
2025

Esophageal cancer staging in Malawi: the feasibility of chest radiography and abdominal ultrasound for initial evaluation.

BMC gastroenterology
2025

In silico analysis of missense SNPs in APPL1 gene: implications on APPL1-AKT2 complex and its relation to MODY 14.

Journal of biomolecular structure &amp; dynamics
2025

Maturity-Onset Diabetes of the Young (MODY) Presenting With a Diabetic Foot Ulcer: A Case Report.

Cureus
2026

Commentary: The heart of the matter: Prioritizing wellness in cardiothoracic training.

The Journal of thoracic and cardiovascular surgery
2026

Current treatment for diabetes: a holistic approach.

Hormones (Athens, Greece)
2026

MODY Is Prevalent in Later-Onset Diabetes and Has Potential for Targeted Therapy but Is Challenging to Identify.

Diabetes
2026

The Importance of Real-World Evidence to Inform RSV Vaccine Guidance.

JAMA internal medicine
2025

Novel Kruppel-like factor 11 variant of maturity-onset diabetes of the young type 7: A case report.

World journal of diabetes
2025

Clinical and cost-effectiveness of non-invasive cell-free DNA testing to optimise foetal outcomes for women with monogenic diabetes due to inactivating glucokinase gene mutations: a case series.

Internal medicine journal
2025

Novel Use of Glucagon-Like Peptide-1 (GLP-1) and Dual Glucose-Dependent Insulinotropic Polypeptide (GIP)/GLP-1 Receptor Agonists in Maturity-Onset Diabetes of the Young (MODY).

Cureus
2026

LoGCC: Local-to-Global Correlation Clustering for Scalar Field Ensembles.

IEEE transactions on visualization and computer graphics
2025

Case Report: NEUROD1 c.-108G>C mutation in a ketosis-prone MODY6 patient: implications for genetic testing and DPP-4 inhibitor therapy.

Frontiers in endocrinology
2026

Advancing pest control: evaluating the effectiveness of RNAi and the BenPol delivery platform for Lucilia cuprina.

Pest management science
2025

Development of production methodologies for scFv-Fc conjugated critical reagents to support CAR-T clinical programs.

Bioanalysis
2025

Gender differences in the distribution of IDL, LDL, and HDL lipoprotein subfractions in MODY compared to type 2 diabetes: Data from the MODY-Ist study.

Journal of diabetes and its complications
2025

Treatment Outcomes of Esophageal Cancer in Malawi: An Analysis of Overall Survival and Patient-Reported Outcomes.

JCO global oncology
2026

Obstructive Sleep Apnea, Resting Heart Rate, and Glycemic Variability in Adults With Maturity-Onset Diabetes of the Young.

Diabetes
2025

Gene Panel-Based Genotyping of 279 Turkish Maturity-Onset Diabetes of the Young Patients from Eastern Anatolia.

Molecular syndromology
2025

Long-Chained Fatty Acid-Based Solid Dispersions of Voriconazole as an Effective Strategy For Achieving Sustained Release.

AAPS PharmSciTech
2025

Earlier Orthopaedic Surgeon Evaluation of Workers' Compensation Associated With Higher Return to Full Duty After Shoulder Arthroscopy.

Journal of the American Academy of Orthopaedic Surgeons. Global research &amp; reviews
2026

Different diabetes types and pancreatic ductal adenocarcinoma: a Mendelian randomization and pathway/gene-set analysis.

Journal of the National Cancer Institute
2026

PDX-1 related diabetes: a case series of two families highlighting challenges in MODY diagnosis.

Acta diabetologica
2025

MODY PDX1P33T: a mouse model reveals phenotypic divergence from human disease.

Frontiers in endocrinology
2025

[Lessons learned from a misdiagnosed case of MODY diabetes: Implications for clinical practice].

Semergen
2026

Additive Effects of Dorzagliatin and Glucagon-Like Peptide 1 Receptor Agonism in a Novel Mouse Model of GCK-MODY and in Obese db/db Mice.

Diabetes
2025

Impact of Prior Ipsilateral Arthrodesis on Subsequent Ankle and Subtalar Fusion Outcomes: A Propensity-Matched Cohort Study.

Foot &amp; ankle international
2025

Breaking Through Barriers: The Transformative Potential of Nanomedicine in Overcoming Drug Delivery Challenges for Psychiatric Disorders.

CNS &amp; neurological disorders drug targets
2026

Glycemia, management and outcomes of pregnant women with maturity-onset diabetes of the young - a single-center case series.

Journal of diabetes and its complications
2026

A Simultaneous Mixed-Methods Examination of Factors Influencing Intent-to-Leave Among Nursing Home Care Providers in 25 Long-Term Care Facilities.

Journal of the American Medical Directors Association
2025

Population prevalence, penetrance, and mortality for genetically confirmed MODY.

The Journal of clinical endocrinology and metabolism
2025

Caloric Restriction Substantially Improves Glucose Regulation in Mice With Hnf1a-Deficient Beta-Cells.

Acta physiologica (Oxford, England)
2025

Sleep patterns in adults and children with less common forms of diabetes.

Frontiers in endocrinology
2025

Rare BLK, CEL, KLF11, PDX1, and PAX4 Gene Variants in Russian Patients with Monogenic Diabetes: Clinical and Molecular Characterization.

Biomedicines
2025

Beyond the Cup: Coffee Extracts as Modulators of Periodontal Inflammation and Bone Remodeling.

Current issues in molecular biology
2026

Spontaneous Ventriculoperitoneal Shunt Extrusion in a Child With Recurrent Meningitis.

Clinical pediatrics
2025

Coexistence of T1DM and GCK-MODY: Case Report and Literature Review.

Journal of clinical research in pediatric endocrinology
2025

Multisociety guidance for infection prevention and control in nursing homes.

Infection control and hospital epidemiology
2026

Online Digital Health Intervention-Safe and Scalable.

JAMA internal medicine
2025

HNF1α-Q125ter-mediated mitochondrial dysfunction and impaired mitophagy in β-cells.

Journal of molecular endocrinology
2025

Assessment of selective salivary properties and taste perception in subjects with oral submucous fibrosis - A case-control study.

Journal of oral biology and craniofacial research
2025

Enhancing the diagnostic yield of monogenic diabetes in unresolved cases with early-onset hyperglycemia.

Communications medicine
2025

Transitioning adolescents with rare forms of diabetes to adult care: challenges and perspectives.

Endocrine connections
2025

Toward Data-Driven MODY Panels: Strong Evidence for NEUROD1 and PDX1, Limited Support for APPL1 and WFS1.

Diabetes
2025

Comparative evaluation of demineralized freeze-dried bone allograft with and without doxycycline hyclate loaded proniosomal gel in the treatment of class II furcation defects: a randomized controlled trial.

Clinical oral investigations
2025

GCK Mutation Analysis and Clinical Profiles of Chinese Pediatric Patients with MODY2: Insights into Screening and Diagnosis.

Pediatric health, medicine and therapeutics
2025

Diabetes mellitus HNF4A-MODY in children from the Russian population: clinical and genetic features.

Frontiers in endocrinology
2025

Swiss Diabetes and Technology recommendations.

Swiss medical weekly
2025

Pediatric PET/MRI: Imaging Techniques, Indications, and Clinical Implementation.

Radiographics : a review publication of the Radiological Society of North America, Inc
2025

Maturity-onset diabetes of the young due to HNF1β variants (HNF1β-MODY): a 2-year follow-up study of six patients from a single diabetes center.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Artificial Intelligence and Clinical Care: JAMA Internal Medicine Call for Papers.

JAMA internal medicine
2025

Antibody-Positive Type 1 Diabetes in a Family With a Pathogenic HNF1A-MODY Variant and Variable Age of Onset.

JCEM case reports
2025

The effect of COVID-19 and sex differences on natural killer cell cytotoxicity.

Frontiers in cellular and infection microbiology
2025

Case Report: Misdiagnosis of Maturity-Onset Diabetes of the Young as type 1, type 2 or gestational diabetes: insights from a Latin American tertiary center.

Frontiers in medicine
2025

A Case of Amyotrophic Lateral Sclerosis With Coexisting Maturity-onset Diabetes of the Young Type 5.

JCEM case reports
2025

Chromosomal instability degrades developmental phenotypes essential for anti-GD2 immunotherapy outcomes in high-risk neuroblastoma.

Cell reports. Medicine
2025

Investigating the Hemorheological, Metabolic, and Physical Performance Effect of a Core Muscle Strengthening Training Program.

Life (Basel, Switzerland)
2025

Accelerating antibody development: sequence and structure-based models for predicting developability properties via size exclusion chromatography.

mAbs
2025

I-BET151 modulates glucokinase gene expression and beta cell function in part through changes in FOXO1 expression.

Diabetologia
2025

Exhausted-like effector CD8 T cells mediate immune-stromal interactions at mucosal Chronic Graft-versus-Host Disease onset.

bioRxiv : the preprint server for biology
2025

Genotype-Phenotype Discrepancies in Family Members With a Novel Glucokinase Mutation: Insights Into GCK-MODY and Its Interplay With Insulin Resistance.

Diabetes
2025

Predicting CSR authenticity in healthcare organizations: an integrated SEM-IPMA-ANN approach through the lens of self-compassion theory.

Journal of health organization and management
2025

Improving Risk Prediction in Complex High-Risk Percutaneous Coronary Intervention: Addressing Missing Data, Calibration, and Clinical Utility.

The Canadian journal of cardiology
2025

MODY: Strategies for a unique form of diabetes.

Journal of education and health promotion
2025

Development of a Chinese-Specific Clinical Model to Predict Maturity-Onset Diabetes of the Young.

Diabetes/metabolism research and reviews
2025

Diazoxide and Continuous Glucose Monitoring as Treatment in a Neonate with Hyperinsulinemic Hypoglycemia due to HNF4A Mutation.

Journal of clinical research in pediatric endocrinology
2025

Sulphonylurea efficacy and end-organ outcomes in the management of HNF4A-MODY.

Diabetic medicine : a journal of the British Diabetic Association
2025

Circulating Tumor DNA and Tissue Testing for Pancreatobiliary Tumors.

JAMA network open
2025

Enhanced guidance on artificial intelligence for medical publication and communication professionals.

Current medical research and opinion
2025

Enzyme-Activated Sugar-Coated Bifunctional Degraders.

Journal of the American Chemical Society
2025

Amorphization of Albendazole Using Organic Acid-based Solid Dispersions for Improved Dissolution: A Solvent-free Approach.

AAPS PharmSciTech
2025

Bridging the Gap Between Training and Reality: A Dental Scientist Trainee Perspective.

Journal of dental education
2025

Common genetic variants modify disease risk and clinical presentation in monogenic diabetes.

Nature metabolism
2025

Cortical versus hippocampal network dysfunction in a human brain assembloid model of epilepsy and intellectual disability.

Cell reports
2025

Endocrinologist-led genomic testing for monogenic diabetes in adult diabetes clinics: a feasibility and outcome study.

Diabetes research and clinical practice
2025

Skilled Nursing Facility Population Characteristics and Length of Stay: A Prospective Cohort Study.

Journal of the American Medical Directors Association
2025

Tailored Therapies for Hereditary Diabetes: Unraveling the Genetic Underpinnings of MODY and Neonatal Diabetes.

Current gene therapy
2025

Identifying care gaps along the HIV treatment failure cascade: A multistate analysis of viral load monitoring, re-suppression, and regimen switches in Zambia.

PLoS medicine
2025

Vericiguat in patients with chronic heart failure and reduced ejection fraction (VICTOR): a double-blind, placebo-controlled, randomised, phase 3 trial.

Lancet (London, England)
2025

Vericiguat for patients with heart failure and reduced ejection fraction across the risk spectrum: an individual participant data analysis of the VICTORIA and VICTOR trials.

Lancet (London, England)
2025

Methodological and Statistical Concerns Regarding "Can Sodium-Glucose Co-Transporter-2 Inhibitors Improve Sleep Quality, Anxiety, and Quality of Life in Patients With Heart Failure?".

Clinical cardiology
2025

Torsades de Pointes Following Massive Transfusion Protocol: Case Highlighting Electrolyte Disturbances After Blood Product Administration.

JACC. Case reports
2025

[Analysis of a family with maturity-onset diabetes of the young type 6 due to a mutation in the NEUROD1 gene].

Zhonghua yi xue za zhi
2026

Genetic and Clinical Characteristics of Monogenic Diabetes in Japan: A Nationwide Study by the Japan Diabetes Society.

The Journal of clinical endocrinology and metabolism
2025

Cost-Effectiveness Thresholds for Venous Thromboembolism Prophylaxis in Ankle Fracture Surgery: A Break-Even Analysis.

Foot &amp; ankle orthopaedics
2025

Feasibility, Acceptability, and Utility of Remote Patient-Reported Outcomes Monitoring in Patients With Lung Cancer: A Moovcare© Study.

Clinical lung cancer
2025

Early-onset diabetes with low utilization of lipid as an energy source carrying a rare missense mutation in the CEL gene.

Endocrinology, diabetes &amp; metabolism case reports
2025

Exciting Discovery of a New Maturity-onset Diabetes of the Young Subtype from India (MODY 15).

The Journal of the Association of Physicians of India
2025

Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort.

Journal of the National Cancer Institute
2025

Oral microbiome alterations after cancer treatment: a scoping review and analysis.

Medical oncology (Northwood, London, England)
2025

Mutational landscapes of HNF MODY gene products display a wide distribution with functional implications.

Endocrine connections
2025

Sources of Racial Disparities in Critical Care Outcomes: A Single Center Analysis.

Journal of health care for the poor and underserved
2025

Maturity onset diabetes of the young and beyond: the changing face of single-gene diabetes.

European journal of endocrinology
2025

Maturity-Onset Diabetes of the Young Associated With a Pathogenic ABCC8 Variant: Expanding the Phenotypic Spectrum.

AACE endocrinology and diabetes
2025

Call for Standardization of C-Peptide Measurement.

Journal of diabetes science and technology
2025

Clinical, laboratory and molecular features of glycogen storage disease type 1a and 1b patients from Turkey: novel mutations and phenotypes.

European journal of pediatrics
2025

Rare Variants in NEUROD1 and PDX1 Are Low-Penetrance Causes of MODY, Whereas Those in APPL1 and WFS1 Are Not Associated With MODY.

Diabetes
2025

Unmasking Diabetes in a Young Adult Male: A Case Report.

Cureus
2025

Predictive value of IL-6, IL-1β, TNF-α, and vaginal pH in diagnosing vaginal microbial infections: A host-inflammatory axis perspective.

Journal of microbiological methods
2025

HNF1A and A1CF coordinate a beta cell transcription-splicing axis that is disrupted in type 2 diabetes.

Cell metabolism
2025

RFX6 heterozygous frame-shift variation causes maturity-onset diabetes mellitus of the young (MODY) with refractory hyperlipidemia: a case report.

AME case reports
2025

Socializing the Benefits of Entering Long-Term Care.

JAMA internal medicine
2026

Children With Diabetes and At Least One Non-Autoimmune Feature Should Be Considered for Monogenic Diabetes Testing.

The Journal of clinical endocrinology and metabolism
2025

Early-Onset Diabetes in Ghana's Upper East Region-Insights From Hospital Data.

Endocrinology, diabetes &amp; metabolism
2025

Supporting Families in the Adult Intensive Care Unit With High Touch-Low Tech Interventions.

JAMA internal medicine
2025

Genetic and clinical characteristics of children with mody: insights into novel HNF4A variants and genotype-phenotype correlation.

Irish journal of medical science
2025

Mitigating Trastuzumab-Doxorubicin Cardiotoxicity With Multiscale Quantitative Systems Toxicology and PBPK-Toxicodynamic Predictive Modeling Framework.

CPT: pharmacometrics &amp; systems pharmacology
2025

TearCare system versus cyclosporine ophthalmic emulsion for the treatment of moderate-to-severe meibomian gland disease associated dry eye disease in the United States: a cost-utility analysis.

Expert review of pharmacoeconomics &amp; outcomes research
2025

Global perspectives on monogenic forms of diabetes.

Diabetologia
Ver todos os 752 no EuropePMC

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Doenças relacionadas

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Referências e fontes

Bases de dados externas citadas neste artigo

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. Development of a Calculator for HNF1A- and HNF4A-MODY in Asian Indians.
    Journal of diabetes science and technology· 2026· PMID 41871534mais citado
  2. Discovery and preclinical activity of the menin-KMT2A inhibitor ziftomenib in acute leukemia models.
    Blood· 2026· PMID 41849234mais citado
  3. Clinical Characterization of Atypical Diabetes: Insights from the GENEPEDIAB Study into the Spectrum Between Type 1 and Monogenic Diabetes.
    Cells· 2026· PMID 41827917mais citado
  4. Efficacy and Tolerability of Topotecan/Cyclophosphamide/Dinutuximab in Relapsed and Refractory High-Risk Neuroblastoma: A Multi-Institutional Retrospective Study.
    Pediatric blood &amp; cancer· 2026· PMID 41823156mais citado
  5. A customized CNN model for signature authentication-Forensic implications.
    Medicine, science, and the law· 2026· PMID 41686745mais citado
  6. Insights from maturity-onset diabetes of the young into impaired insulin secretion in type 2 diabetes.
    Endocr J· 2026· PMID 41987393recente
  7. Prediction of maturity-onset diabetes of the young subtypes using machine learning.
    Front Digit Health· 2026· PMID 41970522recente
  8. Impact of Genetic Testing in Endocrinology Practice: A Single-Centre Experience Focused on Lipid and Monogenic Diabetes Disorders.
    Endocr Pract· 2026· PMID 41966453recente
  9. The conundrum in diagnosing Maturity-Onset Diabetes of the Young (MODY) in a large German pedigree with early-onset diabetes and a novel HNF1A variant.
    Mol Cell Pediatr· 2026· PMID 41961439recente
  10. A worldwide perspective on clinical characteristics and treatment of youth with monogenic diabetes in the SWEET registry.
    J Endocr Soc· 2026· PMID 41958866recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:552(Orphanet)
  2. OMIM OMIM:606391(OMIM)
  3. MONDO:0018911(MONDO)
  4. GARD:3697(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Artigo Wikipedia(Wikipedia)
  8. Q663041(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

Síndrome MODY
Compêndio · Raras BR

Síndrome MODY

ORPHA:552 · MONDO:0018911
Prevalência
1-5 / 10 000
Herança
Autosomal dominant, Not applicable
CID-10
E34.8 · Outros transtornos endócrinos especificados
CID-11
Início
Adolescent, Adult, Childhood
Prevalência
0.0 (Europe)
MedGen
UMLS
C3888631
EuropePMC
Wikidata
Wikipedia
Papers 10a
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