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Holoprosencefalia
ORPHA:2162CID-10 · Q04.2CID-11 · LA05.2DOENÇA RARA

A Holoprosencefalia (HPE) é uma alteração complexa na formação do cérebro que acontece por causa de uma divisão incompleta do prosencéfalo (a parte inicial do cérebro), entre o 18º e o 28º dia de gestação. Isso afeta tanto a parte da frente do cérebro quanto o rosto, resultando em problemas neurológicos e características faciais diferentes, com gravidade que pode variar.

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Introdução

O que você precisa saber de cara

📋

A Holoprosencefalia (HPE) é uma alteração complexa na formação do cérebro que acontece por causa de uma divisão incompleta do prosencéfalo (a parte inicial do cérebro), entre o 18º e o 28º dia de gestação. Isso afeta tanto a parte da frente do cérebro quanto o rosto, resultando em problemas neurológicos e características faciais diferentes, com gravidade que pode variar.

Publicações científicas
2.183 artigos
Último publicado: 1993

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
Antenatal
+ neonatal
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q04.2
🇧🇷Dados SUS / DATASUS
PROCEDIMENTOS SIGTAP (5)
0202010503
Cariótipo — bandas G, Q ou Rgenetic_test
0202010600
Pesquisa de microdeleções/microduplicações por FISHlab_test
0202010694
Sequenciamento completo do exoma (WES)rehabilitation
0202010260
Dosagem de alfa-fetoproteína
0301070040
Atendimento em reabilitação — doenças raras
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Sinais e sintomas

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

Partes do corpo afetadas

😀
Face
61 sintomas
🧠
Neurológico
30 sintomas
📏
Crescimento
23 sintomas
🦴
Ossos e articulações
22 sintomas
👁️
Olhos
15 sintomas
❤️
Coração
13 sintomas

+ 111 sintomas em outras categorias

Características mais comuns

90%prev.
Holoprosencefalia
Muito frequente (99-80%)
90%prev.
Fenda labial bilateral
Muito frequente (99-80%)
90%prev.
Morfologia anormal do sistema nervoso
Muito frequente (99-80%)
90%prev.
Fenda labial mediana
Muito frequente (99-80%)
90%prev.
Incisivo maxilar mediano único
Muito frequente (99-80%)
90%prev.
Fenda palatina mediana
Muito frequente (99-80%)
320sintomas
Muito frequente (7)
Frequente (22)
Ocasional (64)
Sem dados (227)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 320 características clínicas mais associadas, ordenadas por frequência.

HoloprosencefaliaHoloprosencephaly
Muito frequente (99-80%)90%
Fenda labial bilateralBilateral cleft lip
Muito frequente (99-80%)90%
Morfologia anormal do sistema nervosoAbnormal nervous system morphology
Muito frequente (99-80%)90%
Fenda labial medianaMedian cleft lip
Muito frequente (99-80%)90%
Incisivo maxilar mediano únicoSingle median maxillary incisor
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico2.183PubMed
Últimos 10 anos200publicações
Pico202348 papers
Linha do tempo
2026Hoje · 2026🧪 1999Primeiro ensaio clínico📈 2023Ano 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

20 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive, Multigenic/multifactorial, Not applicable, Oligogenic, X-linked dominant.

DISP1Protein dispatched homolog 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Functions in hedgehog (Hh) signaling. Regulates the release and extracellular accumulation of cholesterol-modified hedgehog proteins and is hence required for effective production of the Hh signal (By similarity). Synergizes with SCUBE2 to cause an increase in SHH secretion (PubMed:22902404)

LOCALIZAÇÃO

Membrane

MECANISMO DE DOENÇA

Holoprosencephaly 10

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE10 inheritance pattern is autosomal recessive. Autosomal dominant inheritance with incomplete penetrance or oligogenic inheritance have been reported in some families.

EXPRESSÃO TECIDUAL(Ubíquo)
Pulmão
17.4 TPM
Nervo tibial
16.3 TPM
Cólon sigmoide
15.6 TPM
Testículo
13.9 TPM
Esôfago - Junção
11.0 TPM
OUTRAS DOENÇAS (6)
holoprosencephaly 10alobar holoprosencephalyobsolete midline interhemispheric variant of holoprosencephalylobar holoprosencephaly
HGNC:19711UniProt:Q96F81
SUFUSuppressor of fused homologCandidate gene tested inAltamente restrito
FUNÇÃO

Negative regulator in the hedgehog/smoothened signaling pathway (PubMed:10559945, PubMed:10564661, PubMed:10806483, PubMed:12068298, PubMed:12975309, PubMed:15367681, PubMed:22365972, PubMed:24217340, PubMed:24311597, PubMed:27234298, PubMed:28965847). Down-regulates GLI1-mediated transactivation of target genes (PubMed:15367681, PubMed:24217340, PubMed:24311597). Down-regulates GLI2-mediated transactivation of target genes (PubMed:24217340, PubMed:24311597). Part of a corepressor complex that a

LOCALIZAÇÃO

CytoplasmNucleus

VIAS BIOLÓGICAS (4)
GLI3 is processed to GLI3R by the proteasomeHedgehog 'off' stateDegradation of GLI1 by the proteasomeDegradation of GLI2 by the proteasome
MECANISMO DE DOENÇA

Medulloblastoma

Malignant, invasive embryonal tumor of the cerebellum with a preferential manifestation in children.

EXPRESSÃO TECIDUAL(Ubíquo)
Skin Sun Exposed Lower leg
19.1 TPM
Testículo
17.1 TPM
Skin Not Sun Exposed Suprapubic
16.1 TPM
Nervo tibial
15.3 TPM
Útero
14.5 TPM
OUTRAS DOENÇAS (11)
Joubert syndrome 32basal cell nevus syndrome 2familial multiple meningiomamedulloblastoma with extensive nodularity
HGNC:16466UniProt:Q9UMX1
FGFR1Fibroblast growth factor receptor 1Candidate gene tested inAltamente restrito
FUNÇÃO

Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of embryonic development, cell proliferation, differentiation and migration. Required for normal mesoderm patterning and correct axial organization during embryonic development, normal skeletogenesis and normal development of the gonadotropin-releasing hormone (GnRH) neuronal system. Phosphorylates PLCG1, FRS2, GAB1 and SHB. Ligand binding leads to the activati

LOCALIZAÇÃO

Cell membraneNucleusCytoplasm, cytosolCytoplasmic vesicle

VIAS BIOLÓGICAS (2)
Epithelial-Mesenchymal Transition (EMT) during gastrulationFormation of paraxial mesoderm
MECANISMO DE DOENÇA

Pfeiffer syndrome

A syndrome characterized by the association of craniosynostosis, broad and deviated thumbs and big toes, and partial syndactyly of the fingers and toes. Three subtypes are known: mild autosomal dominant form (type 1); cloverleaf skull, elbow ankylosis, early death, sporadic (type 2); craniosynostosis, early demise, sporadic (type 3).

EXPRESSÃO TECIDUAL(Ubíquo)
Aorta
144.8 TPM
Ovário
142.9 TPM
Artéria tibial
134.1 TPM
Fallopian Tube
122.3 TPM
Cérebro - Hemisfério cerebelar
122.0 TPM
OUTRAS DOENÇAS (20)
Hartsfield-Bixler-Demyer syndromeencephalocraniocutaneous lipomatosisosteoglophonic dysplasiaPfeiffer syndrome
HGNC:3688UniProt:P11362
STILSCL-interrupting locus proteinCandidate gene tested inTolerante
FUNÇÃO

Immediate-early gene. Plays an important role in embryonic development as well as in cellular growth and proliferation; its long-term silencing affects cell survival and cell cycle distribution as well as decreases CDK1 activity correlated with reduced phosphorylation of CDK1. Plays a role as a positive regulator of the sonic hedgehog pathway, acting downstream of PTCH1 (PubMed:16024801, PubMed:9372240). Plays an important role in the regulation of centriole duplication. Required for the onset o

LOCALIZAÇÃO

Cytoplasm, cytosolCytoplasm, cytoskeleton, microtubule organizing center, centrosome, centrioleCytoplasm, cell cortex

EXPRESSÃO TECIDUAL(Tecido-específico)
Linfócitos
21.9 TPM
Testículo
9.8 TPM
Fibroblastos
5.8 TPM
Esôfago - Mucosa
3.5 TPM
Intestino delgado
2.9 TPM
OUTRAS DOENÇAS (7)
microcephaly 7, primary, autosomal recessiveautosomal recessive primary microcephalyalobar holoprosencephalyprecursor T-cell acute lymphoblastic leukemia
HGNC:10879UniProt:Q15468
CRIPTOProtein CriptoCandidate gene tested inTolerante
FUNÇÃO

GPI-anchored cell membrane protein involved in Nodal signaling. Cell-associated CRIPTO acts as a Nodal coreceptor in cis. Shedding of CRIPTO by TMEM8A modulates Nodal signaling by allowing soluble CRIPTO to act as a Nodal coreceptor on other cells (PubMed:27881714). Could play a role in the determination of the epiblastic cells that subsequently give rise to the mesoderm (PubMed:11909953)

LOCALIZAÇÃO

Cell membraneSecreted

VIAS BIOLÓGICAS (3)
Signaling by NODALRegulation of signaling by NODALPOU5F1 (OCT4), SOX2, NANOG activate genes related to proliferation
OUTRAS DOENÇAS (5)
microform holoprosencephalyobsolete midline interhemispheric variant of holoprosencephalyalobar holoprosencephalylobar holoprosencephaly
HGNC:11701UniProt:P13385
FOXH1Forkhead box protein H1Candidate gene tested inTolerante
FUNÇÃO

Transcriptional activator. Recognizes and binds to the DNA sequence 5'-TGT[GT][GT]ATT-3'. Required for induction of the goosecoid (GSC) promoter by TGF-beta or activin signaling. Forms a transcriptionally active complex containing FOXH1/SMAD2/SMAD4 on a site on the GSC promoter called TARE (TGF-beta/activin response element)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (4)
Germ layer formation at gastrulationFormation of axial mesodermSignaling by ActivinSignaling by NODAL
EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
1.6 TPM
Cólon transverso
1.1 TPM
Intestino delgado
0.8 TPM
Pituitária
0.7 TPM
Cerebelo
0.6 TPM
OUTRAS DOENÇAS (5)
alobar holoprosencephalyobsolete midline interhemispheric variant of holoprosencephalyobsolete septopreoptic holoprosencephalymicroform holoprosencephaly
HGNC:3814UniProt:O75593
GAS1Growth arrest-specific protein 1Candidate gene tested inModerado
FUNÇÃO

Specific growth arrest protein involved in growth suppression. Blocks entry to S phase. Prevents cycling of normal and transformed cells. Binds 20(S)-hydroxycholesterol (20(S)-OHC) (By similarity)

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (2)
Activation of SMOLigand-receptor interactions
EXPRESSÃO TECIDUAL(Ubíquo)
Útero
191.2 TPM
Fallopian Tube
164.2 TPM
Adipose Visceral Omentum
112.1 TPM
Ovário
94.2 TPM
Fibroblastos
88.8 TPM
OUTRAS DOENÇAS (5)
lobar holoprosencephalyobsolete septopreoptic holoprosencephalyobsolete midline interhemispheric variant of holoprosencephalyalobar holoprosencephaly
HGNC:4165UniProt:P54826
NODALNodal homologCandidate gene tested inRestrito
FUNÇÃO

Essential for mesoderm formation and axial patterning during embryonic development

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
Signaling by NODALRegulation of signaling by NODAL
MECANISMO DE DOENÇA

Heterotaxy, visceral, 5, autosomal

An autosomal dominant form of visceral heterotaxy, a complex disorder due to disruption of the normal left-right asymmetry of the thoracoabdominal organs. Visceral heterotaxy or situs ambiguus results in randomization of the placement of visceral organs, including the heart, lungs, liver, spleen, and stomach. The organs are oriented randomly with respect to the left-right axis and with respect to one another. It can be associated with a variety of congenital defects including cardiac malformations. HTX5 clinical features include situs inversus viscerum or situs ambiguus, congenital heart defect, transposition of the great vessels ventricular septal defect, atrial septal defect, truncus communis, and dextrocardia.

EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
3.4 TPM
Cerebelo
2.8 TPM
Cervix Ectocervix
2.3 TPM
Cérebro - Hemisfério cerebelar
2.2 TPM
Skin Sun Exposed Lower leg
2.0 TPM
OUTRAS DOENÇAS (8)
heterotaxy, visceral, 5, autosomalmicroform holoprosencephalyvisceral heterotaxyalobar holoprosencephaly
HGNC:7865UniProt:Q96S42
FGF8Fibroblast growth factor 8Candidate gene tested inModerado
FUNÇÃO

Plays an important role in the regulation of embryonic development, cell proliferation, cell differentiation and cell migration. Required for normal brain, eye, ear and limb development during embryogenesis. Required for normal development of the gonadotropin-releasing hormone (GnRH) neuronal system (PubMed:16384934, PubMed:16597617, PubMed:8663044). Plays a role in neurite outgrowth in hippocampal cells (PubMed:21576111)

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Formation of the posterior neural plate
MECANISMO DE DOENÇA

Hypogonadotropic hypogonadism 6 with or without anosmia

A disorder characterized by absent or incomplete sexual maturation by the age of 18 years, in conjunction with low levels of circulating gonadotropins and testosterone and no other abnormalities of the hypothalamic-pituitary axis. In some cases, it is associated with non-reproductive phenotypes, such as anosmia, cleft palate, and sensorineural hearing loss. Anosmia or hyposmia is related to the absence or hypoplasia of the olfactory bulbs and tracts. Hypogonadism is due to deficiency in gonadotropin-releasing hormone and probably results from a failure of embryonic migration of gonadotropin-releasing hormone-synthesizing neurons. In the presence of anosmia, idiopathic hypogonadotropic hypogonadism is referred to as Kallmann syndrome, whereas in the presence of a normal sense of smell, it has been termed normosmic idiopathic hypogonadotropic hypogonadism (nIHH).

EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
1.2 TPM
Córtex cerebral
0.6 TPM
Linfócitos
0.6 TPM
Brain Frontal Cortex BA9
0.6 TPM
Brain Anterior cingulate cortex BA24
0.5 TPM
OUTRAS DOENÇAS (8)
hypogonadotropic hypogonadism 6 with or without anosmiamicroform holoprosencephalyKallmann syndromehypogonadotropic hypogonadism
HGNC:3686UniProt:P55075
DLL1Delta-like protein 1Candidate gene tested inAltamente restrito
FUNÇÃO

Transmembrane ligand protein of NOTCH1, NOTCH2 and NOTCH3 receptors that binds the extracellular domain (ECD) of Notch receptor in a cis and trans fashion manner (PubMed:11006133). Following transinteraction, ligand cells produce mechanical force that depends of a clathrin-mediated endocytosis, requiring ligand ubiquitination, EPN1 interaction, and actin polymerisation; these events promote Notch receptor extracellular domain (NECD) transendocytosis and triggers Notch signaling through induction

LOCALIZAÇÃO

Apical cell membraneCell junction, adherens junctionMembrane raft

VIAS BIOLÓGICAS (10)
Differentiation of Keratinocytes in Interfollicular Epidermis in Mammalian SkinNOTCH2 Activation and Transmission of Signal to the NucleusActivated NOTCH1 Transmits Signal to the NucleusConstitutive Signaling by NOTCH1 PEST Domain MutantsSomitogenesis
MECANISMO DE DOENÇA

Neurodevelopmental disorder with non-specific brain abnormalities and with or without seizures

An autosomal dominant disorder characterized by developmental delay, intellectual disability, seizures, autism spectrum disorder, behavioral abnormalities, and variable non-specific brain malformations.

EXPRESSÃO TECIDUAL(Ubíquo)
Baço
97.4 TPM
Próstata
53.9 TPM
Skin Sun Exposed Lower leg
41.0 TPM
Vagina
40.4 TPM
Skin Not Sun Exposed Suprapubic
40.1 TPM
OUTRAS DOENÇAS (7)
neurodevelopmental disorder with nonspecific brain abnormalities and with or without seizuresmicroform holoprosencephalyobsolete midline interhemispheric variant of holoprosencephalylobar holoprosencephaly
HGNC:2908UniProt:O00548
STAG2Cohesin subunit SA-2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Component of cohesin complex, a complex required for the cohesion of sister chromatids after DNA replication. The cohesin complex apparently forms a large proteinaceous ring within which sister chromatids can be trapped. At anaphase, the complex is cleaved and dissociates from chromatin, allowing sister chromatids to segregate. The cohesin complex may also play a role in spindle pole assembly during mitosis

LOCALIZAÇÃO

NucleusChromosomeChromosome, centromere

VIAS BIOLÓGICAS (2)
Establishment of Sister Chromatid CohesionResolution of Sister Chromatid Cohesion
MECANISMO DE DOENÇA

Mullegama-Klein-Martinez syndrome

An X-linked neurodevelopmental disorder with variable features including intellectual deficiency, microcephaly, microtia, hearing loss, developmental delay, dysmorphic features, language delay, congenital heart defect, and clinodactyly of the 5th finger.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
49.9 TPM
Ovário
38.0 TPM
Fibroblastos
36.7 TPM
Cervix Endocervix
36.4 TPM
Útero
35.4 TPM
OUTRAS DOENÇAS (4)
Mullegama-Klein-Martinez syndromeholoprosencephaly 13, X-linkedXq25 microduplication syndromealobar holoprosencephaly
HGNC:11355UniProt:Q8N3U4
GLI2Zinc finger protein GLI2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Functions as a transcription regulator in the hedgehog (Hh) pathway (PubMed:18455992, PubMed:26565916). Functions as a transcriptional activator (PubMed:19878745, PubMed:24311597, PubMed:9557682). May also function as transcriptional repressor (By similarity). Requires STK36 for full transcriptional activator activity. Required for normal embryonic development (PubMed:15994174, PubMed:20685856) Involved in the smoothened (SHH) signaling pathway Involved in the smoothened (SHH) signaling pathway

LOCALIZAÇÃO

NucleusCytoplasmCell projection, cilium

VIAS BIOLÓGICAS (2)
Hedgehog 'off' stateHedgehog 'on' state
MECANISMO DE DOENÇA

Holoprosencephaly 9

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE9 is characterized by defective anterior pituitary formation and pan-hypopituitarism, with or without overt forebrain cleavage abnormalities, and holoprosencephaly-like midfacial hypoplasia. HPE9 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Ovário
22.1 TPM
Útero
11.2 TPM
Cervix Endocervix
10.1 TPM
Cervix Ectocervix
9.0 TPM
Fibroblastos
7.9 TPM
OUTRAS DOENÇAS (8)
postaxial polydactyly-anterior pituitary anomalies-facial dysmorphism syndromeholoprosencephaly 9combined pituitary hormone deficiencies, genetic formobsolete midline interhemispheric variant of holoprosencephaly
HGNC:4318UniProt:P10070
PTCH1Protein patched homolog 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Acts as a receptor for sonic hedgehog (SHH), indian hedgehog (IHH) and desert hedgehog (DHH). Associates with the smoothened protein (SMO) to transduce the hedgehog's proteins signal. Seems to have a tumor suppressor function, as inactivation of this protein is probably a necessary, if not sufficient step for tumorigenesis

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (4)
Hedgehog 'on' stateActivation of SMOLigand-receptor interactionsHedgehog 'off' state
MECANISMO DE DOENÇA

Basal cell nevus syndrome 1

A form of basal cell nevus syndrome, a disease characterized by nevoid basal cell carcinomas and developmental abnormalities such as rib and craniofacial alterations, polydactyly, syndactyly, and spina bifida. In addition, the patients suffer from a multitude of tumors like fibromas of the ovaries and heart, cysts of the skin, jaws and mesentery, as well as medulloblastomas and meningiomas. BCNS1 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Cérebro - Hemisfério cerebelar
35.1 TPM
Cerebelo
34.9 TPM
Nervo tibial
33.6 TPM
Testículo
17.2 TPM
Bladder
9.1 TPM
OUTRAS DOENÇAS (11)
basal cell carcinoma, susceptibility to, 1holoprosencephaly 7basal cell nevus syndrome 1obsolete septopreoptic holoprosencephaly
HGNC:9585UniProt:Q13635
CNOT1CCR4-NOT transcription complex subunit 1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Scaffolding component of the CCR4-NOT complex which is one of the major cellular mRNA deadenylases and is linked to various cellular processes including bulk mRNA degradation, miRNA-mediated repression, translational repression during translational initiation and general transcription regulation. Additional complex functions may be a consequence of its influence on mRNA expression. Its scaffolding function implies its interaction with the catalytic complex module and diverse RNA-binding proteins

LOCALIZAÇÃO

Cytoplasm, P-bodyNucleus

VIAS BIOLÓGICAS (3)
TP53 regulates transcription of additional cell cycle genes whose exact role in the p53 pathway remain uncertainDeadenylation of mRNAM-decay: degradation of maternal mRNAs by maternally stored factors
MECANISMO DE DOENÇA

Holoprosencephaly 12 with or without pancreatic agenesis

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE12 clinical features include abnormal forebrain development, dysmorphic features, global developmental delay, learning difficulties, and congenital absence of the pancreas in most patients, resulting in early-onset insulin-dependent diabetes mellitus. Other features may include hearing loss and absence of the gallbladder. HPE12 inheritance is autosomal dominant.

OUTRAS DOENÇAS (4)
holoprosencephaly 12 with or without pancreatic agenesisVissers-Bodmer syndromecomplex neurodevelopmental disorderpancreatic agenesis-holoprosencephaly syndrome
HGNC:7877UniProt:A5YKK6
SHHSonic hedgehog proteinDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

The C-terminal part of the sonic hedgehog protein precursor displays an autoproteolysis and a cholesterol transferase activity (By similarity). Both activities result in the cleavage of the full-length protein into two parts (ShhN and ShhC) followed by the covalent attachment of a cholesterol moiety to the C-terminal of the newly generated ShhN (By similarity). Both activities occur in the endoplasmic reticulum (By similarity). Once cleaved, ShhC is degraded in the endoplasmic reticulum (By simi

LOCALIZAÇÃO

Endoplasmic reticulum membraneGolgi apparatus membraneSecretedCell membrane

VIAS BIOLÓGICAS (5)
Hedgehog 'on' stateActivation of SMOLigand-receptor interactionsRelease of Hh-Np from the secreting cellFormation of lateral plate mesoderm
MECANISMO DE DOENÇA

Microphthalmia/Coloboma 5

A disorder of eye formation, ranging from small size of a single eye to complete bilateral absence of ocular tissues. Ocular abnormalities like opacities of the cornea and lens, scaring of the retina and choroid, and other abnormalities may also be present. Ocular colobomas are a set of malformations resulting from abnormal morphogenesis of the optic cup and stalk, and the fusion of the fetal fissure (optic fissure).

EXPRESSÃO TECIDUAL(Tecido-específico)
Nervo tibial
21.7 TPM
Glândula adrenal
7.7 TPM
Fígado
7.4 TPM
Estômago
3.8 TPM
Rim - Medula
3.5 TPM
OUTRAS DOENÇAS (15)
microphthalmia, isolated, with coloboma 5holoprosencephaly 3solitary median maxillary central incisor syndrometibia, hypoplasia or aplasia of, with polydactyly
HGNC:10848UniProt:Q15465
SIX3Homeobox protein SIX3Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcriptional regulator which can act as both a transcriptional repressor and activator by binding a ATTA homeodomain core recognition sequence on these target genes. During forebrain development represses WNT1 expression allowing zona limitans intrathalamica formation and thereby ensuring proper anterio-posterior patterning of the diencephalon and formation of the rostral diencephalon. Acts as a direct upstream activator of SHH expression in the rostral diencephalon ventral midline and that i

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Holoprosencephaly 2

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE2 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Tecido-específico)
Brain Nucleus accumbens basal ganglia
52.0 TPM
Pituitária
42.5 TPM
Brain Caudate basal ganglia
41.0 TPM
Brain Putamen basal ganglia
30.8 TPM
Hipotálamo
13.7 TPM
OUTRAS DOENÇAS (8)
holoprosencephaly 2schizencephalyacquired schizencephalyalobar holoprosencephaly
HGNC:10889UniProt:O95343
ZIC2Zinc finger protein ZIC 2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Acts as a transcriptional activator or repressor. Plays important roles in the early stage of organogenesis of the CNS. Activates the transcription of the serotonin transporter SERT in uncrossed ipsilateral retinal ganglion cells (iRGCs) to refine eye-specific projections in primary visual targets. Its transcriptional activity is repressed by MDFIC. Involved in the formation of the ipsilateral retinal projection at the optic chiasm midline. Drives the expression of EPHB1 on ipsilaterally project

LOCALIZAÇÃO

NucleusCytoplasm

VIAS BIOLÓGICAS (1)
Formation of the anterior neural plate
MECANISMO DE DOENÇA

Holoprosencephaly 5

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE5 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
298.1 TPM
Cerebelo
248.3 TPM
Córtex cerebral
9.4 TPM
Brain Anterior cingulate cortex BA24
8.5 TPM
Brain Frontal Cortex BA9
7.4 TPM
OUTRAS DOENÇAS (6)
holoprosencephaly 5microform holoprosencephalyalobar holoprosencephalyobsolete midline interhemispheric variant of holoprosencephaly
HGNC:12873UniProt:O95409
PLCH11-phosphatidylinositol 4,5-bisphosphate phosphodiesterase eta-1Disease-causing germline mutation(s) inRestrito
FUNÇÃO

The production of the second messenger molecules diacylglycerol (DAG) and inositol 1,4,5-trisphosphate (IP3) is mediated by calcium-activated phosphatidylinositol-specific phospholipase C enzymes

LOCALIZAÇÃO

CytoplasmMembrane

VIAS BIOLÓGICAS (1)
Synthesis of IP3 and IP4 in the cytosol
MECANISMO DE DOENÇA

Holoprosencephaly 14

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE14 inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Tecido-específico)
Testículo
24.3 TPM
Cérebro - Hemisfério cerebelar
5.9 TPM
Tireoide
5.7 TPM
Cerebelo
5.7 TPM
Brain Frontal Cortex BA9
5.7 TPM
OUTRAS DOENÇAS (2)
holoprosencephaly 14alobar holoprosencephaly
HGNC:29185UniProt:Q4KWH8
TGIF1Homeobox protein TGIF1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Binds to a retinoid X receptor (RXR) responsive element from the cellular retinol-binding protein II promoter (CRBPII-RXRE). Inhibits the 9-cis-retinoic acid-dependent RXR alpha transcription activation of the retinoic acid responsive element. Active transcriptional corepressor of SMAD2. Links the nodal signaling pathway to the bifurcation of the forebrain and the establishment of ventral midline structures. May participate in the transmission of nuclear signals during development and in the adu

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (2)
Downregulation of SMAD2/3:SMAD4 transcriptional activitySMAD2/SMAD3:SMAD4 heterotrimer regulates transcription
MECANISMO DE DOENÇA

Holoprosencephaly 4

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE4 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
29.3 TPM
Fallopian Tube
21.4 TPM
Próstata
18.7 TPM
Pulmão
18.4 TPM
Skin Not Sun Exposed Suprapubic
18.3 TPM
OUTRAS DOENÇAS (6)
holoprosencephaly 4microform holoprosencephalyobsolete septopreoptic holoprosencephalylobar holoprosencephaly
HGNC:11776UniProt:Q15583
CDONCell adhesion molecule-related/down-regulated by oncogenesDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Component of a cell-surface receptor complex that mediates cell-cell interactions between muscle precursor cells. Promotes differentiation of myogenic cells (By similarity)

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (3)
Activation of SMOLigand-receptor interactionsMyogenesis
MECANISMO DE DOENÇA

Holoprosencephaly 11

A form of holoprosencephaly, a structural anomaly of the brain in which the developing forebrain fails to correctly separate into right and left hemispheres. It is a genetically and clinically heterogeneous disorder with a wide spectrum of severity, ranging from alobar holoprosencephaly with severe facial abnormalities, such as cyclopia and proboscis, to mild forms that include lobar or microform holoprosencephaly, without cerebral malformations and with mild craniofacial defects. HPE11 inheritance is autosomal dominant.

OUTRAS DOENÇAS (7)
holoprosencephaly 11alobar holoprosencephalypituitary stalk interruption syndromelobar holoprosencephaly
HGNC:17104UniProt:Q4KMG0

Variantes genéticas (ClinVar)

1,947 variantes patogênicas registradas no ClinVar.

🧬 DISP1: GRCh37/hg19 1q21.1-44(chr1:143932350-249224684)x3 ()
🧬 DISP1: GRCh37/hg19 1q41(chr1:218253812-223932158)x1 ()
🧬 DISP1: NM_001377229.1(DISP1):c.2064C>A (p.Cys688Ter) ()
🧬 DISP1: DISP1, 1-BP DEL, NT4408 ()
🧬 DISP1: DISP1, GLY1047SER ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 2,900 variantes classificadas pelo ClinVar.

145
1305
1450
Patogênica (5.0%)
VUS (45.0%)
Benigna (50.0%)
VARIANTES MAIS SIGNIFICATIVAS
ZIC2: NM_007129.5(ZIC2):c.1021_1028del (p.Gly341fs) [Likely pathogenic]
SIX3: NM_005413.4(SIX3):c.124_141dup (p.Gly47_Ser48insGlyGlyAlaGlyGlyGly) [Uncertain significance]
CDON: NM_001378964.1(CDON):c.1073A>T (p.His358Leu) [Uncertain significance]
FOXH1: NM_003923.3(FOXH1):c.799G>A (p.Gly267Arg) [Uncertain significance]
SHH: NM_000193.4(SHH):c.907G>A (p.Ala303Thr) [Uncertain significance]

Vias biológicas (Reactome)

91 vias biológicas associadas aos genes desta condição.

Degradation of GLI1 by the proteasome Degradation of GLI2 by the proteasome GLI3 is processed to GLI3R by the proteasome Hedgehog 'off' state Hedgehog 'on' state PI3K Cascade PIP3 activates AKT signaling Signaling by FGFR1 amplification mutants Signaling by activated point mutants of FGFR1 FGFR1b ligand binding and activation FGFR1c ligand binding and activation FGFR1c and Klotho ligand binding and activation Constitutive Signaling by Aberrant PI3K in Cancer NCAM signaling for neurite out-growth Signal transduction by L1 Phospholipase C-mediated cascade: FGFR1 Downstream signaling of activated FGFR1 SHC-mediated cascade:FGFR1 PI-3K cascade:FGFR1 FRS-mediated FGFR1 signaling Negative regulation of FGFR1 signaling Signaling by FGFR1 in disease RAF/MAP kinase cascade PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling Signaling by plasma membrane FGFR1 fusions Epithelial-Mesenchymal Transition (EMT) during gastrulation Formation of paraxial mesoderm Phosphorylated Orc1 is ubiquitinated while still associated with chromatin Signaling by NODAL Regulation of signaling by NODAL POU5F1 (OCT4), SOX2, NANOG activate genes related to proliferation Signaling by Activin Germ layer formation at gastrulation Formation of axial mesoderm Ligand-receptor interactions Activation of SMO Signaling by activated point mutants of FGFR3 FGFR4 ligand binding and activation FGFR3b ligand binding and activation FGFR3c ligand binding and activation FGFR2c ligand binding and activation Activated point mutants of FGFR2 Phospholipase C-mediated cascade; FGFR2 Phospholipase C-mediated cascade; FGFR3 Phospholipase C-mediated cascade; FGFR4 PI-3K cascade:FGFR2 SHC-mediated cascade:FGFR2 FRS-mediated FGFR2 signaling SHC-mediated cascade:FGFR3 FRS-mediated FGFR3 signaling PI-3K cascade:FGFR3 FRS-mediated FGFR4 signaling SHC-mediated cascade:FGFR4 PI-3K cascade:FGFR4 Negative regulation of FGFR2 signaling Activated NOTCH1 Transmits Signal to the Nucleus Constitutive Signaling by NOTCH1 PEST Domain Mutants Constitutive Signaling by NOTCH1 t(7;9)(NOTCH1:M1580_K2555) Translocation Mutant Constitutive Signaling by NOTCH1 HD Domain Mutants Constitutive Signaling by NOTCH1 HD+PEST Domain Mutants NOTCH2 Activation and Transmission of Signal to the Nucleus NOTCH3 Activation and Transmission of Signal to the Nucleus MECP2 regulates transcription of neuronal ligands Differentiation of Keratinocytes in Interfollicular Epidermis in Mammalian Skin Somitogenesis Nephron development Meiotic synapsis Separation of Sister Chromatids Establishment of Sister Chromatid Cohesion Cohesin Loading onto Chromatin Resolution of Sister Chromatid Cohesion SUMOylation of DNA damage response and repair proteins Estrogen-dependent gene expression GLI proteins bind promoters of Hh responsive genes to promote transcription RUNX2 regulates chondrocyte maturation Class B/2 (Secretin family receptors) Deadenylation of mRNA TP53 regulates transcription of additional cell cycle genes whose exact role in the p53 pathway remain uncertain M-decay: degradation of maternal mRNAs by maternally stored factors Hedgehog ligand biogenesis Hh mutants are degraded by ERAD Release of Hh-Np from the secreting cell HHAT G278V doesn't palmitoylate Hh-Np Formation of lateral plate mesoderm Developmental Lineage of Multipotent Pancreatic Progenitor Cells Transcription of NOTCH2NLB gene Formation of the anterior neural plate Synthesis of IP3 and IP4 in the cytosol Downregulation of SMAD2/3:SMAD4 transcriptional activity SMAD2/SMAD3:SMAD4 heterotrimer regulates transcription Myogenesis

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Publicações mais relevantes

Timeline de publicações
579 papers (10 anos)
#1

Vertical inheritance and unique differential phenotypes of reciprocal recombinant chromosome 18 within a multi-generation family.

European journal of human genetics : EJHG2026 Jan

Carriers of balanced pericentric inversions are at risk for producing unbalanced gametes because of meiotic recombination resulting in de novo deletion and duplication of distal chromosome ends. Recombinant chromosomes generally lead to significant imbalances resulting in anomalous clinical phenotypes in offspring, hence they are typically not inherited. Therefore, the vertical transmission of recombinant chromosomes is a clinically rare event. Using genomic microarray and karyotyping, we describe inheritance of recombinant chromosomes in a three-generation family with the grandmother carrying a mosaic pericentric inversion of chromosome 18. Three children inherited the balanced inversion and one child with a mild phenotype inherited a de novo recombinant chromosome 18. In the third generation, a newborn with a variant of holoprosencephaly inherited an unmodified recombinant chromosome from her mother. Despite having the same karyotype predicting loss of the TGIF1 gene from the 18p terminus, the mother exhibits a relatively unaffected phenotype. The cousin of the child with holoprosencephaly carries the reciprocal recombinant chromosome 18 with a much milder phenotype. We verified the cytogenetic mechanism and corresponding clinical phenotypes in affected individuals and illustrated possible recombinant chromosome consequences of the inversion of chromosome 18 in this three-generation family.

#2

A Rare Case Report on Holoprosencephaly With Cyclopia: Jimma University Medical Center, Ethiopia.

Case reports in obstetrics and gynecology2026

Holoprosencephaly is a congenital brain malformation resulting from incomplete division of the forebrain (prosencephalon) into two hemispheres during early embryonic development, typically during the 4th-6th week of gestation. Cyclopia is a rare and fatal form of holoprosencephaly characterized by severe craniofacial abnormalities resulting from incomplete cleavage of the embryonic prosencephalon, leading to the failure of separation of the orbital cavities. The prevalence is 1.31 per 10,000 for live births and 1:250 for spontaneous abortions. This is a case report of a 33-year-old Gravida 5, Para 4 woman with no history of abortion and a prior history of normal vaginal deliveries, who presented to the obstetric triage with a 7-day history of absent fetal movements. After a detailed history was taken, a physical examination and ultrasound scan confirmed an intrauterine fetal death. Termination of the pregnancy was decided after the mother was counseled on the possible outcome of the pregnancy. Induction with misoprostol per the hospital protocol was given to deliver an 1800-g, Grade 3 macerated male fetus with a diagnosis of a severe form of holoprosencephaly with cyclopia and proboscis. Although holoprosencephaly is rare, early first-trimester anatomical ultrasound conducted by an experienced clinician is critical for accurate diagnosis, and evaluation of severity is pivotal for counseling families regarding expected outcomes and pregnancy termination decisions.

#3

Cyclopia: Facial deformity indicating severe holoprosencephaly with imaging findings of brain: A case report.

Radiology case reports2026 Apr

Holoprosencephaly results from incomplete separation of the cerebral hemispheres. Cyclopia is a facial manifestation of Holoprosencephaly, characterized by a midline single orbit and proboscis. Prenatal diagnosis is done by ultrasonography and can be supplemented by MRI. This case uniquely presents ultrasound and gross morphology correlation of cyclopia in a resource limited setting, and a correlation between facial features, intra-cranial anatomy, and lifespan. We report a 39-year-old gravida 3 para 2 woman with a history of alcohol consumption who presented for her first antenatal checkup at 19 weeks of gestation. Ultrasound revealed a single lateral ventricle with fused thalami, absent orbital structures, and a midline cystic protrusion (proboscis). After thorough counseling, pregnancy was terminated, and cyclopia was confirmed on gross inspection. Karyotyping and fetal echocardiography were not done due to parental preference. Alobar holoprosencephaly, the most severe subtype, is diagnosed by identification of single ventricle and associated facial anomalies. Lobar holoprosencephaly, however, requires coronal imaging to demonstrate absence of cavum septum pellucidum and fusion of frontal horns. The severity of facial anomalies correlates with underlying brain malformations. Chromosomal anomalies, maternal diabetes, infections, and teratogenic exposures like alcohol are known risk factors. Differential diagnoses include proboscis lateralis, midline encephaloceles and frontonasal dysplasia. This case highlights the prognostic significance of facial and brain anomalies and diagnostic utility of prenatal ultrasound in diagnosing holoprosencephaly underscoring the necessity of timely prenatal visits. Definitive genomic studies were not feasible, but maternal alcohol consumption can be considered as a possible risk factor.

#4

Phenotypic Spectrum and Chromosomal Discordance in Alobar Holoprosencephaly: A Comparative Case Series from a Tertiary Referral Center.

International medical case reports journal2026

Holoprosencephaly (HPE) is a rare congenital malformation of the forebrain caused by incomplete midline cleavage, often accompanied by craniofacial abnormalities. The condition arises from multifactorial etiologies, including genetic, environmental, and maternal factors. Early prenatal diagnosis is essential for parental counseling, management decisions, and detection of associated anomalies. We report two cases of alobar HPE diagnosed in the third trimester by ultrasonography. The first case involved a 41-year-old primigravida at 32 weeks of gestation, with ultrasound findings of fused thalami, single ventricle, microcephaly, and hypotelorism. The fetus was delivered with severe midline craniofacial abnormalities and a normal female karyotype (46,XX). The second case involved a 41-year-old multiparous woman at 32 weeks of gestation. Ultrasound revealed fused thalami, absent falx cerebri and corpus callosum, and a proboscis. The infant presented with synophthalmia and proboscis, and karyotyping confirmed trisomy 13. Both pregnancies were terminated after counseling, and postnatal findings confirmed the prenatal diagnosis. This case series demonstrates the phenotypic and chromosomal variability within alobar HPE and underscores the diagnostic value of detailed ultrasonography combined with genetic analysis. Late detection in the third trimester limited reproductive options and highlights the importance of improved anomaly screening pathways. Early diagnosis remains crucial for comprehensive parental counselling and perinatal planning.

#5

Cyclopia, A Rare and Lethal Congenital Anomaly: Report of Two Cases, 2025.

International medical case reports journal2026

Cyclopia is an extremely rare and fatal congenital anomaly in which causes are not commonly known. This condition is marked by the failure of the eye orbits to properly divide, resulting in a single eye field or closely positioned eye fields. There is no specific treatment, and the prognosis is generally poor with most babies dying shortly after birth or during infancy. These two newborns were born to multiparous mothers, which died soon after birth. Diagnosis was made postpartum with centrally located single eye and long proboscis on the midline of the forehead, with the second had associated with cystic hygroma. Both cases had no other gross visible congenital anomalies on the extremities, abdomen, and head. But other anomalies as renal, central nervous system, cardiac and chromosomal anomalies cannot be excluded. Both cases had died immediately, and health professionals should experience with detailed antenatal ultrasound scanning to detect these congenital anomalies antepartum for better counseling of patients. Routine antenatal ultrasound assessment is crucial in low resource countries where ultrasound scanning is commonly done in higher health facilities.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC1.101 artigos no totalmostrando 197

2026

A Rare Case Report on Holoprosencephaly With Cyclopia: Jimma University Medical Center, Ethiopia.

Case reports in obstetrics and gynecology
2026

Cyclopia: Facial deformity indicating severe holoprosencephaly with imaging findings of brain: A case report.

Radiology case reports
2026

Phenotypic Spectrum and Chromosomal Discordance in Alobar Holoprosencephaly: A Comparative Case Series from a Tertiary Referral Center.

International medical case reports journal
2026

Cyclopia, A Rare and Lethal Congenital Anomaly: Report of Two Cases, 2025.

International medical case reports journal
2026

From Multiple Congenital Anomalies to Pituitary Gland Malformation: Wide Spectrum of Clinical Features in a Family With FOXA2 Variant.

American journal of medical genetics. Part A
2026

Radiogenomics of congenital brain malformations: Linking embryology, genetics, and imaging.

Neuroradiology
2026

The 129S1/SvImJ Mouse Strain Is Resistant to the Effects of Early Embryonic Alcohol and Other Sonic Hedgehog Inhibitors.

Birth defects research
2025

Digital Technologies in Diagnosing Solitary Median Maxillary Central Incisor Syndrome.

Children (Basel, Switzerland)
2025

Malformation Pattern and Molecular Findings in the FGFR1-Related Hartsfield Syndrome Phenotype.

Medical sciences (Basel, Switzerland)
2026

Novel variants in STAG2 and PKD1 associate with multiple congenital malformations and autosomal dominant polycystic kidney disease in a Chinese family: A case report and literature review.

Experimental and therapeutic medicine
2025

Congenital Anomalies in a Neonate With Partial Monosomy of Chromosome 21 q Arm: A Case Report.

Cureus
2026

The Role of the Hedgehog Pathway in Alcohol-Induced Birth Defects.

Advances in experimental medicine and biology
2026

Surgeon Perspectives on Cleft Lip and Palate Repair in Patients With Life-Limiting and Terminal Illnesses: An ACPA Member Survey.

The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
2025

Congenital Syngnathia With Holoprosencephaly: A Case Report of a Fatal Presentation in a Resource-Limited Setting.

Clinical case reports
2026

The hidden brain in cleft: Clinical presentation in patients with median cerebrofacial dysgenesis.

Journal of plastic, reconstructive &amp; aesthetic surgery : JPRAS
2026

Undetected cases after implementation of first-trimester anomaly scan in low-risk population: insights from the IMITAS study.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2025

Prerepair Mortality in Cleft Lip and/or Palate: A Retrospective Analysis of Early Childhood Deaths.

The Cleft palate-craniofacial journal : official publication of the American Cleft Palate-Craniofacial Association
2025

Management of Prenatally Diagnosed Malformations of the Central Nervous System: Factors Influencing Decision-making and the Time of Termination of Pregnancy.

Geburtshilfe und Frauenheilkunde
2025

Ophthalmologic Findings in an Induced Model of Holoprosencephaly in Zebrafish.

The Journal of comparative neurology
2025

Recent advances in the diagnosis and molecular pathogenesis of holoprosencephaly: a review.

Journal of applied genetics
2025

13q Deletion Syndrome Presenting with Lymphopenia Detected Through Newborn Screening for Primary Immunodeficiencies.

International journal of molecular sciences
2025

A Case of Ethmocephaly in a Clomiphene Citrate Induced Pregnancy: A Case Report and Literature Review.

Clinical case reports
2026

Multidisciplinary orthodontic and prosthetic treatment of an eleven-year-old patient with a solitary median maxillary central incisor: A case report.

International orthodontics
2025

De novo inherited Xq25 deletion: hints from preimplantation genetic testing in alobar holoprosencephaly.

European journal of obstetrics, gynecology, and reproductive biology
2025

Exome sequencing uncovers promising candidate genes for foetal structural malformations.

The Indian journal of medical research
2025

Sublingual Administration of Desmopressin Oral Disintegrating Tablet in a Neonate With Central Diabetes Insipidus.

Cureus
2026

Prenatal Diagnosis of Hartsfield Syndrome in the Fetus With Isolated Ectrodactyly Caused by a Novel Variant in FGFR1.

American journal of medical genetics. Part A
2025

Centriolar protein PIBF1 is required for craniofacial and forebrain development.

Developmental biology
2025

Prenatal imaging diagnosis of iniencephaly apertus associated with heterotaxy syndrome, alobar holoprosencephaly and myelomeningocele: a case report.

AJOG global reports
2025

Cyclopia Syndrome with Neck Presentation: A Case of Alobar Holoprosencephaly and Prenatal Diagnostic Challenges.

International medical case reports journal
2025

Hypernatremia, Hyperlipemia and Hemorrhagic Enteritis in a Hypodipsic Dog with Corpus Callosum Dysplasia.

Animals : an open access journal from MDPI
2026

Vertical inheritance and unique differential phenotypes of reciprocal recombinant chromosome 18 within a multi-generation family.

European journal of human genetics : EJHG
2025

Holoprosencephaly and cyclopia in bmp7b and bmpr1ba Crispant zebrafish.

Animal cells and systems
2025

Fibroblast growth factor 8: Multifaceted role in development and developmental disorder.

Genes &amp; diseases
2025

Origin stories of neural crest roles in craniofacial development: A tale of the meninges.

Differentiation; research in biological diversity
2025

Surgical Nuances in Ultrasound-Guided Percutaneous Distal Catheter Placement in Pediatric Ventriculoatrial Shunts.

Cureus
2025

Plumbagin, a natural derivative of 1,4-naphthoquinone, induces cyclopic phenomenon via increased apoptosis and ROS generation in the early stage of zebrafish embryos.

Ecotoxicology and environmental safety
2025

Hypersynchronous EEG Patterns in a Patient with Holoprosencephaly.

Clinical EEG and neuroscience
2025

Partial monosomy 18p and 21q due to a paternal reciprocal translocation leading to holoprosencephaly.

Human genome variation
2025

Insights into holoprosencephaly using multimodal high-resolution imaging and 3D histology.

Journal of neuropathology and experimental neurology
2025

Effects of alcohol on the transcriptome, methylome and metabolome of in vitro gastrulating human embryonic cells.

Disease models &amp; mechanisms
2025

Loss of Asxl1 disrupts telencephalic midline integrity through dysregulation of SIX3 target genes.

Biochemical and biophysical research communications
2025

Successful management of human parainfluenza virus-3 outbreak in a tertiary neonatal intensive care unit.

Saudi medical journal
2025

Developmental, Endocrine, and Ophthalmologic Outcomes in Children Prenatally Diagnosed With Midline Brain Malformations.

Pediatric neurology
2025

Functional properties of the γ-ENaC-A635V mutation in a patient with severe hyponatremia.

Hormones (Athens, Greece)
2025

Congenital external hydrocephalus: A rare presentation of lobar holoprosencephaly in a neonate.

Radiology case reports
2025

Situs Inversus in an Infant With Hypomandibular Faciocranial Syndrome: Clinical Overlap With the Agnathia-Otocephaly Complex.

American journal of medical genetics. Part A
2025

Sonographic diagnosis of fetal eye anomalies and their association with syndromal diseases: A retrospective multicenter analysis of 264 cases.

Acta obstetricia et gynecologica Scandinavica
2024

Detailed Analysis of Fetal Malformations of the Supratentorial Structures of the Brain in High-Risk Pregnancies at 12-14 Gestational Weeks by Transvaginal 3D Ultrasound Examination.

Ultrasound international open
2025

Lobar holoprosencephaly with associated meningocele: A rare case report of a 25-year-old patient with multiple seizures.

Radiology case reports
2025

Complete pentalogy of Cantrell associated with ectopia cordis and multiple anomalies: A case report from a low-resource setting.

Radiology case reports
2025

Sonic Hedgehog Determines Early Retinal Development and Adjusts Eyeball Architecture.

International journal of molecular sciences
2024

Hydranencephaly in a Newborn: A Case Report and a Review of the Literature.

Cureus
2025

Solitary median maxillary central incisor syndrome caused by 22q11.2 microdeletion.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2024

Intracranial hemorrhage and additional anomalies detected on prenatal magnetic resonance imaging: A large, retrospective study in two tertiary medical institutions.

Heliyon
2025

Alobar holoprosencephaly with cyclopia and proboscis.

Pediatric radiology
2024

Alobar Holoprosencephaly in a Newborn: A Case Report of Prenatal Diagnosis and a Review of the Literature.

Cureus
2025

Embryotoxicity of statins and other prescribed drugs with reported off-target effects on cholesterol biosynthesis.

Reproductive toxicology (Elmsford, N.Y.)
2025

Mesencephalosynapsis and aqueductal stenosis.

Journal of neuropathology and experimental neurology
2025

Fetal Phenotyping and Whole Exome Sequencing for 12 Egyptian Families With Serine Biosynthesis Defect: Novel Clinical and Allelic Findings With a Founder Effect.

Prenatal diagnosis
2025

Prenatal ultrasound assisted diagnosis of de-novo terminal 7q deletion syndrome: A case report with literature review.

Radiology case reports
2025

Prenatally Diagnosed Holoprosencephaly: Review of the Literature and Practical Recommendations for Pediatric Neurologists.

Pediatric neurology
2024

MRI Evaluation of Corpus Callosum Malformation and Associated Anomalies: A Retrospective Cross-Sectional Study.

Cureus
2024

The Role of Antenatal Ultrasound Scans in the Early Detection of Alobar Holoprosencephaly: A Case Report.

Cureus
2024

Corpus callosal agenesis with gray matter heterotopia and bilateral eye coloboma in an infant: A case report.

Radiology case reports
2024

Efcab7 deletion sensitizes mice to the teratogenic effects of gastrulation-stage alcohol exposure.

Reproductive toxicology (Elmsford, N.Y.)
2025

Biallelic TXNDC15 variants associated with Joubert syndrome-related molar tooth sign and forebrain malformation.

Journal of human genetics
2025

Holoprosencephaly spectrum: an up-to-date overview of classification, genetics and neuroimaging.

Japanese journal of radiology
2025

Loss-of-Function Variant in PPP1R12A -Related Urogenital and/or Brain Malformation Syndrome: Expanded Phenotype of Sex Reversal.

American journal of medical genetics. Part A
2025

Congenital Pyriform Aperture Stenosis: Not All Patients Require Open Repair.

Otolaryngology--head and neck surgery : official journal of American Academy of Otolaryngology-Head and Neck Surgery
2024

ARID1A-BAF coordinates ZIC2 genomic occupancy for epithelial-to-mesenchymal transition in cranial neural crest specification.

American journal of human genetics
2024

CNOT1 p.Arg535Cys variant in holoprosencephaly with late onset diabetes mellitus.

American journal of medical genetics. Part A
2025

Introduction of a nationwide first-trimester anomaly scan in the Dutch national screening program.

American journal of obstetrics and gynecology
2024

Prenatal detection of distal 18p deletion by chromosomal microarray analysis: Three case reports and literature review.

Medicine
2024

Pulsatile gonadotropin-releasing hormone therapy induces spermatogenesis in pituitary stalk interruption syndrome: A case report and review of the literature.

World journal of clinical cases
2024

Dual white matter pathology in fetal holoprosencephaly featuring concurrent malformative and destructive features: A case series.

Journal of neuropathology and experimental neurology
2023

Phenotypic and cytogenetic variability of patau syndrome in Morocco.

African health sciences
2024

CDON Mutation Related to Nose Deformity with Variable Expression in Holoprosencephaly in an Iranian Family: A Case Report.

Iranian journal of public health
2024

Prevalence of congenital anomalies and prenatal diagnosis by birth institution (public vs. non-public): indicators of inequality in access to elective termination of pregnancy for fetal anomalies.

Journal of community genetics
2024

Knockout mice with pituitary malformations help identify human cases of hypopituitarism.

Genome medicine
2024

Human Malformed Perinatal Anthropological Crania Contribute to New Insight in the Extension of Bone Malformations in Cranial Development.

Fetal and pediatric pathology
2024

Effects of GLP1 receptor analogues in obesity with neurodevelopmental disorder: case report of a patient with holoprosencephaly.

European journal of clinical nutrition
2024

Use of an Orthodontic and Otolaryngological Approach in an Infant with Holoprosencephaly.

Children (Basel, Switzerland)
2024

Classification of isolated versus multiple birth defects: An automated process for population-based registries.

American journal of medical genetics. Part A
2024

Prenatal identification of a pathogenic maternal FGFR1 variant in two consecutive pregnancies with fetal forebrain malformations.

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
2024

Post-mortem rapid aneuploidy testing for holoprosencephaly.

Birth defects research
2024

Truncating variants of the sterol recognition region of SHH cause hypertelorism phenotype rather than hypotelorism-holoprosencephaly.

American journal of medical genetics. Part A
2024

Impact of Sonic Hedgehog-dependent sphenoid bone defect on craniofacial growth.

Clinical and experimental dental research
2024

Detection of non-cardiac fetal abnormalities on ultrasound at 11-14 weeks: systematic review and meta-analysis.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2024

DISP1 deficiency: Monoallelic and biallelic variants cause a spectrum of midline craniofacial malformations.

Genetics in medicine : official journal of the American College of Medical Genetics
2024

Cheiloplasty for False Median Cleft Lip Associated With Holoprosencephaly: The Use of Skin Graft for Philtrum Reconstruction, the Risk of Simultaneous Columella Reconstruction.

Eplasty
2024

A male fetus with cyclopia was discovered after miscarriage: A rare case report from Syria.

Clinical case reports
2024

Prdm15 acts upstream of Wnt4 signaling in anterior neural development of Xenopus laevis.

Frontiers in cell and developmental biology
2024

Holoprosencephaly: Syndromic or Non-syndromic is the question.

Indian journal of pathology &amp; microbiology
2024

STAG2: Computational Analysis of Missense Variants Involved in Disease.

International journal of molecular sciences
2024

Positive non-invasive prenatal testing for trisomy 13 in the first trimester in a pregnancy with fetal holoprosencephaly, cebocephaly and postaxial polydactyly.

Taiwanese journal of obstetrics &amp; gynecology
2024

Differential alternative splicing analysis links variation in ZRSR2 to a novel type of oral-facial-digital syndrome.

Genetics in medicine : official journal of the American College of Medical Genetics
2023

Exome sequencing in 16 patients with pituitary stalk interruption syndrome: A monocentric study.

PloS one
2023

The rare malformation holoprosencephaly: pathogenesis, association with pregestational diabetes and the possible link with food pollutants.

Annali dell'Istituto superiore di sanita
2023

Prenatal diagnosis of Hartsfield syndrome with a novel genetic variant.

Prenatal diagnosis
2023

Prenatal Magnetic Resonance Imaging helps Discover Cerebellar Dysplasia or Malformations in Foetuses.

Current medical imaging
2023

Comparing Repeatability and Agreement between Commonly Used Corneal Imaging Devices in Keratoconus.

Optometry and vision science : official publication of the American Academy of Optometry
2023

Survival of neonates, infants, and children with birth defects: a population-based study in Texas, 1999-2018.

Lancet regional health. Americas
2024

Whole-genome sequencing analysis in fetal structural anomalies: novel phenotype-genotype discoveries.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2023

Efficacy of tolvaptan in an infant with syndrome of inappropriate antidiuretic hormone secretion associated with holoprosencephaly: A case report.

World journal of clinical cases
2023

Holoprosencephaly (HPE) : case report and review of the literature.

International journal of surgery case reports
2023

Sonic hedgehog signaling in craniofacial development.

Differentiation; research in biological diversity
2023

The utility of balloon dilation for piriform aperture stenosis - A first line surgical treatment.

International journal of pediatric otorhinolaryngology
2022

Congenital complete arhinia with alobar holoprosencephaly.

Ghana medical journal
2023

Semilobar Holoprosencephaly Caused by a Novel and De Novo ZIC2 Pathogenic Variant.

Balkan journal of medical genetics : BJMG
2023

Adult Radiographic Presentation of Corpus Callosal Agenesis With a Single Interhemispheric Cyst and Dandy-Walker Malformation: A Case Report.

Cureus
2023

Cranial vault reduction cranioplasty for severe macrocephaly due to holoprosencephaly and subdural hygroma: a case report.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2023

Biocontrol of Fusarium Species Utilizing Indigenous Rooibos and Honeybush Extracts.

Microbiology spectrum
2023

Holoprosencephaly: Review of Embryology, Clinical Phenotypes, Etiology and Management.

Children (Basel, Switzerland)
2023

Novel sonic hedgehog gene variant in a patient with hyponatremia, microsomia, and midline defects; phenotype description in association with a variant of unknown significance [c.755_757del p.(Phe252del)] and an approach to salt-wasting in SHH-related adrenal disorders.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2023

Holoprosencephaly with a Special Form of Anophthalmia Result from Experimental Induction of bmp4, Oversaturating BMP Antagonists in Zebrafish.

International journal of molecular sciences
2023

Isolated Agnathia-Otocephaly Complex Diagnosed Prenatally for Ex-Utero Intrapartum Treatment: A Case Report.

The American journal of case reports
2023

Schizencephaly diagnosed after an episode of seizure during labor: A case report.

Clinical case reports
2023

The role of routine first-trimester ultrasound screening for central nervous system abnormalities: a longitudinal single-center study using an unselected cohort with 3-year experience.

BMC pregnancy and childbirth
2023

Solitary Median Maxillary Central Incisor in Hartsfield Syndrome: A Case Report.

International journal of clinical pediatric dentistry
2024

Genotype-phenotype analysis of ocular findings in Rubinstein-Taybi syndrome - A case report and review of literature.

Ophthalmic genetics
2023

Role of Fetal Magnetic Resonance Imaging in Differentiating Isolated Septal Agenesis from Septo-Optic Dysplasia: Case Study and Review.

Fetal diagnosis and therapy
2023

Congenital hyporhinia with associated malformations: Case report of a rare congenital anomaly.

Clinical case reports
2023

Brain malformations in diprosopia observed in clinical cases, museum specimens and artistic representations.

Orphanet journal of rare diseases
2023

Holoprosencephaly in Patau Syndrome.

Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao Paulo
2023

Fetal Cyclopia, Proboscis, Holoprosencephaly, and Polydactyly: A Case Report With Review of Literature.

Cureus
2023

Rare manifestations of alobar holoprosencephaly and the potential causes: a report of two cases.

Annals of medicine and surgery (2012)
2023

Mystical and mythological believes not only limited to psychiatric diseases? A dynamic overview of medicine.

Annals of medicine and surgery (2012)
2023

Antenatal and Postnatal Diagnosis of Semilobar Holoprosencephaly: Two Case Reports.

Global pediatric health
2023

Geospatiotemporal and Causal Inferential Study of European Epidemiological Patterns of Cannabis- and Substance-Related Congenital Orofacial Anomalies.

Journal of xenobiotics
2023

A novel missense variant of FGFR1 in a Japanese girl with Kallmann syndrome and holoprosencephaly.

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
2023

Pericentric Inversion of Chromosome 9 in Twins With Cyclopia: A Rare Entity.

Cureus
2023

CNKSR2, a downstream mediator of retinoic acid signaling, modulates the Ras/Raf/MEK pathway to regulate patterning and invagination of the chick forebrain roof plate.

Development (Cambridge, England)
2023

[Value of chromosomal microarray analysis for the diagnosis of fetuses with anomalies of central nervous system].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2023

First Trimester Ultrasound Detection of Fetal Central Nervous System Anomalies.

Brain sciences
2023

TMEM161B regulates cerebral cortical gyration, Sonic Hedgehog signaling, and ciliary structure in the developing central nervous system.

Proceedings of the National Academy of Sciences of the United States of America
2023

Cyclopia in a newborn rhesus macaque born to a dam infected with SIV and receiving antiretroviral therapy during pregnancy.

Current trends in immunology
2023

INDIAMAN-20 (INstant DIAgnosis of 20 Major ANomalies) protocol: application of IOTA diagnostic strategy to fetal anomalies.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2023

Pathogenic/likely pathogenic copy number variations and regions of homozygosity in fetal central nervous system malformations.

Archives of gynecology and obstetrics
2023

An analysis of the incidence, risk factors, and timing of development of cyclops lesions after anterior cruciate ligament reconstruction.

The Knee
2023

Bone-patellar tendon-bone autograft and female sex are associated with the presence of cyclops lesions and syndrome after anterior cruciate ligament reconstruction.

Knee surgery, sports traumatology, arthroscopy : official journal of the ESSKA
2022

Ecce Homo: Moving past labels to lives.

International journal of paleopathology
2023

Evolution in the clinic: Maladaptive units and "minor anomalies".

American journal of medical genetics. Part A
2023

Muscle spasms as presenting feature of Nivelon-Nivelon-Mabile syndrome.

American journal of medical genetics. Part A
2022

Synophtalmia on a newborn.

SAGE open medical case reports
2022

[Syntelencephaly: medial interhemispheric variant of holoprosencephaly].

The Pan African medical journal
2022

Prognostic factors, psychomotor development and life of trisomy 13 patients.

Pediatrics international : official journal of the Japan Pediatric Society
2022

Imaging of Congenital Malformations of the Brain.

Clinics in perinatology
2023

Cyclops lesions associated with both bundles and selective bundle repair of the anterior cruciate ligament.

Acta radiologica (Stockholm, Sweden : 1987)
2022

Antenatal Ultrasonographic Diagnosis of a Constellation of Alobar Holoprosencephaly, Ethmocephaly, and Hydronephrosis in a Case of Early-Onset Intrauterine Growth Retardation: A Case Report.

Cureus
2022

An infant with patau syndrome associated with congenital heart defects.

Annals of medicine and surgery (2012)
2022

A plunging ranula in a child with holoprosencephaly: a case of unique pathophysiology and difficult airway management.

Journal of the Korean Association of Oral and Maxillofacial Surgeons
2022

The diagnosis of the middle interhemispheric variant of holoprosencephaly with fetal MRI.

Radiologia
2022

Sinus pericranii associated with syntelencephaly: a case report.

BMC neurology
2022

Campylobacter jejuni subdural hygroma infection in a 2-year old boy: case report and a brief literature review.

BMC infectious diseases
2023

Heterozygous variants in SIX3 and POU1F1 cause pituitary hormone deficiency in mouse and man.

Human molecular genetics
2022

Non-Invasive Detection of a De Novo Frameshift Variant of STAG2 in a Female Fetus: Escape Genes Influence the Manifestation of X-Linked Diseases in Females.

Journal of clinical medicine
2023

Clinical spectrum of orbital and ocular abnormalities on fetal MRI.

Pediatric radiology
2022

Prenatal diagnosis and pregnancy outcome of major structural anomalies detectable in the first trimester: A population-based cohort study in the Netherlands.

Paediatric and perinatal epidemiology
2022

Vertical course of fetal anterior cerebral artery as clue to prenatal diagnosis of syntelencephaly.

Ultrasound in obstetrics &amp; gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology
2022

Cyclopia baby: Congenital lethal malformation: Rare case report.

International journal of surgery case reports
2022

Fetal Brain Development: Regulating Processes and Related Malformations.

Life (Basel, Switzerland)
2022

Hedgehog Autoprocessing: From Structural Mechanisms to Drug Discovery.

Frontiers in molecular biosciences
2022

Brain Organization and Human Diseases.

Cells
2022

Genomic architecture of fetal central nervous system anomalies using whole-genome sequencing.

NPJ genomic medicine
2022

Fetal Description of the Pancreatic Agenesis and Holoprosencephaly Syndrome Associated to a Specific CNOT1 Variant.

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society
2022

Polymicrogyria, aventriculy, polydactyly, encephalocele, callosal agenesis (PAPEC): a new syndrome?

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2022

Alobar holoprosencephaly with cebocephaly in a neonate: A rare case report from Northern Tanzania.

International journal of surgery case reports
2022

Intracerebral retina-like pigmented tissue in a stillborn fetus with holoprosencephaly.

Clinical neuropathology
2022

Survival of children with rare structural congenital anomalies: a multi-registry cohort study.

Orphanet journal of rare diseases
2022

Successful treatment of hypodipsic/adipsic hypernatremia in a cat with lobar holoprosencephaly using oral desmopressin.

JFMS open reports
2022

Mosaicism in Hartsfield syndrome.

European journal of medical genetics
2022

Familial microdeletion 18p11.32 to 18p11.31 in a Chinese family with normal phenotype.

Molecular cytogenetics
2022

Sonography of fetal holoprosencephaly: a guide to recognize the lesser varieties.

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
2022

Alobar holoprosencephaly: Exploring mothers' perspectives on prenatal decision-making and prognostication.

Prenatal diagnosis
2022

Entomopathogenic nematodes associated with organic honeybush (Cyclopia spp.) cultivation in South Africa.

Journal of helminthology
2022

Application of quantitative fluorescent polymerase chain reaction analysis for the rapid confirmation of trisomy 13 of maternal origin in a pregnancy with fetal holoprosencephaly, cyclopia, polydactyly, omphalocele and cell culture failure.

Taiwanese journal of obstetrics &amp; gynecology
2022

Alobar Holoprosencephaly in an Aborted American Quarter Horse Fetus.

Journal of equine veterinary science
2022

Patterning of the antero-ventral mammalian brain: Lessons from holoprosencephaly comparative biology in man and mouse.

WIREs mechanisms of disease
2022

Risks of specific congenital anomalies in offspring of women with diabetes: A systematic review and meta-analysis of population-based studies including over 80 million births.

PLoS medicine
2022

Final notes on: Response to Galassi et al. concerning the paper by Turgut et al. "Three mythic giants for common fœtal malformation called 'cyclopia': Polyphemus, Tepegöz and Grendel", Child's Nervous System, DOI 10.1007/s00381-019-04,207-y.

Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
2022

Holoprosencephalia, hypoplasia of corpus callosum and cerebral heterotopia in a male belted Galloway heifer with adipsia.

BMC veterinary research
2021

Asymptomatic Hypernatremia in an Infant with Midline Defects.

EJIFCC
2022

Varied presentation of lobar holoprosencephaly as a cause of macrocephaly in a neonate.

BMJ case reports
2021

Concepts in Multifactorial Etiology of Developmental Disorders: Gene-Gene and Gene-Environment Interactions in Holoprosencephaly.

Frontiers in cell and developmental biology
2021

Preterm infant with diprosopus and holoprosencephaly.

Clinical case reports
2021

Zebrafish as a Model for Fetal Alcohol Spectrum Disorders.

Frontiers in pharmacology
2021

Home-Based Monitoring of Eating in Adolescents: A Pilot Study.

Nutrients
2022

Rhomboencephalosynapsis: Review of the Literature.

World neurosurgery
2022

Truncating and zinc-finger variants in GLI2 are associated with hypopituitarism.

American journal of medical genetics. Part A
2021

Case Report: An Infant With Kabuki Syndrome, Alobar Holoprosencephaly and Truncus Arteriosus: A Case for Whole Exome Sequencing in Neonates With Congenital Anomalies.

Frontiers in genetics
2021

Structural Insight into the Binding of TGIF1 to SIN3A PAH2 Domain through a C-Terminal Amphipathic Helix.

International journal of molecular sciences
2021

Rare case of skeletal third class in a subject suffering from Solitary Median Maxillary Central Incisor syndrome (SMMCI) associated to panhypopituitarism.

Head &amp; face medicine
2021

Solitary Median Maxillary Central Incisor: A Case Report with 3-Year Follow-Up and Literature Review.

Contemporary clinical dentistry
2021

Cyclopia, a newborn with a single eye, a rare but lethal congenital anomaly: A case report.

International journal of surgery case reports
2021

Alobar Holoprosencephaly with Cebocephaly in a Neonate Born to an HIV-Positive Mother in Eastern Uganda.

Case reports in otolaryngology
2021

GAS1 is required for NOTCH-dependent facilitation of SHH signaling in the ventral forebrain neuroepithelium.

Development (Cambridge, England)
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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

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. Vertical inheritance and unique differential phenotypes of reciprocal recombinant chromosome 18 within a multi-generation family.
    European journal of human genetics : EJHG· 2026· PMID 40628998mais citado
  2. A Rare Case Report on Holoprosencephaly With Cyclopia: Jimma University Medical Center, Ethiopia.
    Case reports in obstetrics and gynecology· 2026· PMID 41798263mais citado
  3. Cyclopia: Facial deformity indicating severe holoprosencephaly with imaging findings of brain: A case report.
    Radiology case reports· 2026· PMID 41727820mais citado
  4. Phenotypic Spectrum and Chromosomal Discordance in Alobar Holoprosencephaly: A Comparative Case Series from a Tertiary Referral Center.
    International medical case reports journal· 2026· PMID 41710465mais citado
  5. Cyclopia, A Rare and Lethal Congenital Anomaly: Report of Two Cases, 2025.
    International medical case reports journal· 2026· PMID 41710460mais citado
  6. CNOT1-Related Vissers-Bodmer Syndrome.
    · 1993· PMID 41911374recente
  7. Counseling Preferences of Expectant Parents With a Fetal Neurologic Diagnosis: A Scoping Review of the Literature.
    J Child Neurol· 2026· PMID 41885704recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:2162(Orphanet)
  2. MONDO:0016296(MONDO)
  3. GARD:6665(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Artigo Wikipedia(Wikipedia)
  7. Q1459821(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

Holoprosencefalia
Compêndio · Raras BR

Holoprosencefalia

ORPHA:2162 · MONDO:0016296
Prevalência
Unknown
Herança
Autosomal recessive, Multigenic/multifactorial, Not applicable, Oligogenic, X-linked dominant
CID-10
Q04.2 · Holoprosencefalia
CID-11
Início
Antenatal, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C0078982
EuropePMC
Wikidata
Wikipedia
Papers 10a
DiscussaoAtiva

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