Raras
Buscar doenças, sintomas, genes...
Disgenesia mesenquimatosa do segmento anterior do olho, familiar
ORPHA:88632CID-11 · LA11DOENÇA RARA

Espectro de anomalias de desenvolvimento que afetam o desenvolvimento do segmento anterior do globo ocular, resultantes de anormalidades na migração e diferenciação da crista neural durante o desenvolvimento embriológico (síndrome de Axenfeld-Rieger, anomalia de Peters, ceratocone posterior e iridosquise).

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Espectro de anomalias de desenvolvimento que afetam o desenvolvimento do segmento anterior do globo ocular, resultantes de anormalidades na migração e diferenciação da crista neural durante o desenvolvimento embriológico (síndrome de Axenfeld-Rieger, anomalia de Peters, ceratocone posterior e iridosquise).

Pesquisas ativas
1 ensaio
2 total registrados no ClinicalTrials.gov
Publicações científicas
15 artigos
Último publicado: 2023 Oct-Dec
🏥
SUS: Sem cobertura SUSScore: 0%
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Sinais e sintomas

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

Partes do corpo afetadas

👁️
Olhos
42 sintomas
🧠
Neurológico
24 sintomas
😀
Face
19 sintomas
🦴
Ossos e articulações
12 sintomas
👂
Ouvidos
6 sintomas
🫃
Digestivo
5 sintomas

+ 81 sintomas em outras categorias

Características mais comuns

Dedo do pé largo
Hipoplasia cerebelar
Telangiectasia
Cegueira
Hérnia umbilical
Anormalidade da artéria pulmonar
205sintomas
Sem dados (205)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 205 características clínicas mais associadas, ordenadas por frequência.

Dedo do pé largoBroad toe
Hipoplasia cerebelarCerebellar hypoplasia
Telangiectasia
CegueiraBlindness
Hérnia umbilicalUmbilical hernia

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico15PubMed
Últimos 10 anos200publicações
Pico202490 papers
Linha do tempo
2026Hoje · 2026🧪 2006Primeiro ensaio clínico📈 2024Ano 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

10 genes identificados com associação a esta condição.

Autosomal dominant
CPAMD8C3 and PZP-like alpha-2-macroglobulin domain-containing protein 8Disease-causing germline mutation(s) inTolerante
LOCALIZAÇÃO

SecretedCell membrane

MECANISMO DE DOENÇA

Anterior segment dysgenesis 8

A form of anterior segment dysgenesis, a group of defects affecting anterior structures of the eye including cornea, iris, lens, trabecular meshwork, and Schlemm canal. Anterior segment dysgeneses result from abnormal migration or differentiation of the neural crest derived mesenchymal cells that give rise to components of the anterior chamber during eye development. Different anterior segment anomalies may exist alone or in combination, including iris hypoplasia, enlarged or reduced corneal diameter, corneal vascularization and opacity, posterior embryotoxon, corectopia, polycoria, abnormal iridocorneal angle, ectopia lentis, and anterior synechiae between the iris and posterior corneal surface. Clinical conditions falling within the phenotypic spectrum of anterior segment dysgeneses include aniridia, Axenfeld anomaly, Reiger anomaly/syndrome, Peters anomaly, and iridogoniodysgenesis. ASGD8 patients predominantly manifest iris and lens abnormalities, in the absence of retinal abnormalities or extra-ocular features. ASGD8 transmission pattern is consistent with autosomal recessive inheritance.

OUTRAS DOENÇAS (1)
anterior segment dysgenesis 8
HGNC:23228UniProt:Q8IZJ3
PAX6Paired box protein Pax-6Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Transcription factor with important functions in the development of the eye, nose, central nervous system and pancreas. Required for the differentiation of pancreatic islet alpha cells (By similarity). Competes with PAX4 in binding to a common element in the glucagon, insulin and somatostatin promoters. Regulates specification of the ventral neuron subtypes by establishing the correct progenitor domains (By similarity). Acts as a transcriptional repressor of NFATC1-mediated gene expression (By s

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (5)
Synthesis, secretion, and inactivation of Glucagon-like Peptide-1 (GLP-1)Regulation of gene expression in beta cellsActivation of anterior HOX genes in hindbrain development during early embryogenesisFormation of the anterior neural plateSynthesis, secretion, and inactivation of Glucose-dependent Insulinotropic Polypeptide (GIP)
MECANISMO DE DOENÇA

Aniridia 1

A congenital, bilateral, panocular disorder characterized by complete absence of the iris or extreme iris hypoplasia. Aniridia is not just an isolated defect in iris development but it is associated with macular and optic nerve hypoplasia, cataract, corneal changes, nystagmus. Visual acuity is generally low but is unrelated to the degree of iris hypoplasia. Glaucoma is a secondary problem causing additional visual loss over time.

EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
40.8 TPM
Cerebelo
36.9 TPM
Córtex cerebral
3.5 TPM
Brain Caudate basal ganglia
3.4 TPM
Brain Anterior cingulate cortex BA24
3.3 TPM
OUTRAS DOENÇAS (17)
coloboma, ocular, autosomal dominantisolated optic nerve hypoplasiaautosomal dominant keratitisfoveal hypoplasia 1
HGNC:8620UniProt:P26367
ATOH7Transcription factor ATOH7Candidate gene tested inTolerante
FUNÇÃO

Transcription factor that binds to DNA at the consensus sequence 5'-CAG[GC]TG-3' (PubMed:31696227). Dimerization with TCF3 isoform E47 may be required in certain situations (PubMed:31696227). Binds to gene promoters and enhancer elements, and thereby regulates a transcriptional program of retinal ganglion cell (RGC) determinant genes (By similarity). Although the exact mechanism is not certain, retinal transcription regulation by ATOH7 has a role in RGC determination and survival, photoreceptor

LOCALIZAÇÃO

NucleusPerikaryonCell projection, axon

MECANISMO DE DOENÇA

Persistent hyperplastic primary vitreous, autosomal recessive

A developmental eye malformation associated with microphthalmia, cataract, glaucoma, and congenital retinal non-attachment. It is due to failure of the primary vitreous to regress in utero, resulting in the presence of a retrolental fibrovascular membrane with persistence of the posterior portion of the tunica vasculosa lentis and hyaloid artery. Disease manifestations range from a trivial remnant of hyaloid vessels to a dense fibrovascular mass causing lens opacity and retinal detachment.

INTERAÇÕES PROTEICAS (4)
OUTRAS DOENÇAS (3)
persistent hyperplastic primary vitreous, autosomal recessivepersistent hyperplastic primary vitreousanterior segment dysgenesis 7
HGNC:13907UniProt:Q8N100
PITX2Pituitary homeobox 2Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

May play a role in myoblast differentiation. When unphosphorylated, associates with an ELAVL1-containing complex, which stabilizes cyclin mRNA and ensuring cell proliferation. Phosphorylation by AKT2 impairs this association, leading to CCND1 mRNA destabilization and progression towards differentiation Involved in the establishment of left-right asymmetry in the developing embryo

LOCALIZAÇÃO

NucleusCytoplasm

VIAS BIOLÓGICAS (1)
TFAP2 (AP-2) family regulates transcription of other transcription factors
MECANISMO DE DOENÇA

Axenfeld-Rieger syndrome 1

An autosomal dominant disorder of morphogenesis that results in abnormal development of the anterior segment of the eye, and results in blindness from glaucoma in approximately 50% of affected individuals. Additional features include aniridia, maxillary hypoplasia, hypodontia, anal stenosis, redundant periumbilical skin.

EXPRESSÃO TECIDUAL(Tecido-específico)
Bladder
25.4 TPM
Pituitária
18.0 TPM
Músculo esquelético
16.0 TPM
Esôfago - Muscular
10.3 TPM
Esôfago - Junção
4.5 TPM
OUTRAS DOENÇAS (8)
ring dermoid of corneaanterior segment dysgenesis 4Axenfeld-Rieger syndrome type 1Peters anomaly
HGNC:9005UniProt:Q99697
PXDNPeroxidasin homologDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalyzes the two-electron oxidation of bromide by hydrogen peroxide and generates hypobromite as a reactive intermediate which mediates the formation of sulfilimine cross-links between methionine and hydroxylysine residues within an uncross-linked collagen IV/COL4A1 NC1 hexamer (PubMed:18929642, PubMed:19590037, PubMed:22842973, PubMed:25708780, PubMed:25713063, PubMed:27697841, PubMed:28154175, PubMed:34679700). In turns, directly contributes to the collagen IV network-dependent fibronectin/FN

LOCALIZAÇÃO

Secreted, extracellular space, extracellular matrixEndoplasmic reticulumCell surfaceSecreted, extracellular space, extracellular matrix, basement membrane

VIAS BIOLÓGICAS (2)
Regulation of MITF-M-dependent genes involved in extracellular matrix, focal adhesion and epithelial-to-mesenchymal transitionCrosslinking of collagen fibrils
MECANISMO DE DOENÇA

Anterior segment dysgenesis 7

A form of anterior segment dysgenesis, a group of defects affecting anterior structures of the eye including cornea, iris, lens, trabecular meshwork, and Schlemm canal. Anterior segment dysgeneses result from abnormal migration or differentiation of the neural crest derived mesenchymal cells that give rise to components of the anterior chamber during eye development. Different anterior segment anomalies may exist alone or in combination, including iris hypoplasia, enlarged or reduced corneal diameter, corneal vascularization and opacity, posterior embryotoxon, corectopia, polycoria, abnormal iridocorneal angle, ectopia lentis, and anterior synechiae between the iris and posterior corneal surface. Clinical conditions falling within the phenotypic spectrum of anterior segment dysgeneses include aniridia, Axenfeld anomaly, Reiger anomaly/syndrome, Peters anomaly, and iridogoniodysgenesis. ASGD7 is an autosomal recessive disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
169.3 TPM
Aorta
90.5 TPM
Tecido adiposo
67.8 TPM
Adipose Visceral Omentum
62.6 TPM
Ovário
61.1 TPM
OUTRAS DOENÇAS (1)
anterior segment dysgenesis 7
HGNC:14966UniProt:Q92626
FOXE3Forkhead box protein E3Disease-causing germline mutation(s) inDesconhecido
FUNÇÃO

Transcription factor that controls lens epithelial cell growth through regulation of proliferation, apoptosis and cell cycle (PubMed:22527307, PubMed:25504734). During lens development, controls the ratio of the lens fiber cells to the cells of the anterior lens epithelium by regulating the rate of proliferation and differentiation (By similarity). Controls lens vesicle closure and subsequent separation of the lens vesicle from ectoderm (By similarity). Controls the expression of DNAJB1 in a pat

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Anterior segment dysgenesis 2

A form of anterior segment dysgenesis, a group of defects affecting anterior structures of the eye including cornea, iris, lens, trabecular meshwork, and Schlemm canal. Anterior segment dysgeneses result from abnormal migration or differentiation of the neural crest derived mesenchymal cells that give rise to components of the anterior chamber during eye development. Different anterior segment anomalies may exist alone or in combination, including iris hypoplasia, enlarged or reduced corneal diameter, corneal vascularization and opacity, posterior embryotoxon, corectopia, polycoria, abnormal iridocorneal angle, ectopia lentis, and anterior synechiae between the iris and posterior corneal surface. Clinical conditions falling within the phenotypic spectrum of anterior segment dysgeneses include aniridia, Axenfeld anomaly, Reiger anomaly/syndrome, Peters anomaly, and iridogoniodysgenesis. Some ASGD2 patients show congenital primary aphakia, a defect caused by eye development arrest around the 4th-5th week of gestation. This prevents the formation of any lens structure and leads to severe secondary ocular anomalies, including a complete aplasia of the anterior segment of the eye. In contrast, in secondary aphakic eyes, lens induction has occurred, and the lens vesicle has developed to some degree but finally has progressively resorbed perinatally, leading, therefore, to less severe ocular defects. ASGD2 inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
2.3 TPM
Nervo tibial
1.2 TPM
Cérebro - Hemisfério cerebelar
0.6 TPM
Cerebelo
0.6 TPM
Brain Putamen basal ganglia
0.6 TPM
INTERAÇÕES PROTEICAS (3)
OUTRAS DOENÇAS (5)
cataract 34 multiple typescongenital primary aphakiafamilial thoracic aortic aneurysm and aortic dissectionPeters anomaly
HGNC:3808UniProt:Q13461
ITPR1Inositol 1,4,5-trisphosphate-gated calcium channel ITPR1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Inositol 1,4,5-trisphosphate-gated calcium channel that, upon inositol 1,4,5-trisphosphate binding, mediates calcium release from the endoplasmic reticulum (ER) (PubMed:10620513, PubMed:27108797). Undergoes conformational changes upon ligand binding, suggesting structural flexibility that allows the channel to switch from a closed state, capable of interacting with its ligands such as 1,4,5-trisphosphate and calcium, to an open state, capable of transferring calcium ions across the ER membrane (

LOCALIZAÇÃO

Endoplasmic reticulum membraneCytoplasmic vesicle, secretory vesicle membraneCytoplasm, perinuclear region

VIAS BIOLÓGICAS (10)
Ion homeostasisRegulation of insulin secretionFCGR3A-mediated IL10 synthesisAntigen activates B Cell Receptor (BCR) leading to generation of second messengersCLEC7A (Dectin-1) induces NFAT activation
MECANISMO DE DOENÇA

Spinocerebellar ataxia 15

Spinocerebellar ataxia is a clinically and genetically heterogeneous group of cerebellar disorders. Patients show progressive incoordination of gait and often poor coordination of hands, speech and eye movements, due to degeneration of the cerebellum with variable involvement of the brainstem and spinal cord. SCA15 is an autosomal dominant cerebellar ataxia (ADCA). It is very slow progressing form with a wide range of onset, ranging from childhood to adult. Most patients remain ambulatory.

EXPRESSÃO TECIDUAL(Ubíquo)
Artéria tibial
82.0 TPM
Cerebelo
76.2 TPM
Artéria coronária
59.8 TPM
Aorta
52.7 TPM
Fallopian Tube
47.8 TPM
OUTRAS DOENÇAS (3)
aniridia-cerebellar ataxia-intellectual disability syndromespinocerebellar ataxia type 29spinocerebellar ataxia type 15/16
HGNC:6180UniProt:Q14643
CYP1B1Cytochrome P450 1B1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

A cytochrome P450 monooxygenase involved in the metabolism of various endogenous substrates, including fatty acids, steroid hormones and vitamins (PubMed:10681376, PubMed:11555828, PubMed:12865317, PubMed:15258110, PubMed:20972997). Mechanistically, uses molecular oxygen inserting one oxygen atom into a substrate, and reducing the second into a water molecule, with two electrons provided by NADPH via cytochrome P450 reductase (NADPH--hemoprotein reductase) (PubMed:10681376, PubMed:11555828, PubM

LOCALIZAÇÃO

Endoplasmic reticulum membraneMicrosome membraneMitochondrion

VIAS BIOLÓGICAS (3)
Endogenous sterolsSynthesis of (16-20)-hydroxyeicosatetraenoic acids (HETE)Synthesis of epoxy (EET) and dihydroxyeicosatrienoic acids (DHET)
MECANISMO DE DOENÇA

Anterior segment dysgenesis 6

A form of anterior segment dysgenesis, a group of defects affecting anterior structures of the eye including cornea, iris, lens, trabecular meshwork, and Schlemm canal. Anterior segment dysgeneses result from abnormal migration or differentiation of the neural crest derived mesenchymal cells that give rise to components of the anterior chamber during eye development. Different anterior segment anomalies may exist alone or in combination, including iris hypoplasia, enlarged or reduced corneal diameter, corneal vascularization and opacity, posterior embryotoxon, corectopia, polycoria, abnormal iridocorneal angle, ectopia lentis, and anterior synechiae between the iris and posterior corneal surface. Clinical conditions falling within the phenotypic spectrum of anterior segment dysgeneses include aniridia, Axenfeld anomaly, Reiger anomaly/syndrome, Peters anomaly, and iridogoniodysgenesis. ASGD6 patients predominantly manifest Peters anomaly. Peters anomaly consists of corneal leukoma, defects in the posterior structures of the cornea such as absence of the posterior corneal stroma and Descemet membrane, and a variable degree of iridocorneal and/or keratolenticular adhesions. Over 50% of patients develop glaucoma in childhood.

EXPRESSÃO TECIDUAL(Ubíquo)
Nervo tibial
100.0 TPM
Cervix Endocervix
66.1 TPM
Fibroblastos
62.9 TPM
Fallopian Tube
58.5 TPM
Útero
53.7 TPM
OUTRAS DOENÇAS (5)
glaucoma 3Aanterior segment dysgenesis 6CYP1B1-related glaucoma with or without anterior segment dysgenesisjuvenile open angle glaucoma
HGNC:2597UniProt:Q16678
PITX3Pituitary homeobox 3Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Transcriptional regulator which is important for the differentiation and maintenance of meso-diencephalic dopaminergic (mdDA) neurons during development. In addition to its importance during development, it also has roles in the long-term survival and maintenance of the mdDA neurons. Activates NR4A2/NURR1-mediated transcription of genes such as SLC6A3, SLC18A2, TH and DRD2 which are essential for development of mdDA neurons. Acts by decreasing the interaction of NR4A2/NURR1 with the corepressor

LOCALIZAÇÃO

Nucleus

MECANISMO DE DOENÇA

Anterior segment dysgenesis 1

A form of anterior segment dysgenesis, a group of defects affecting anterior structures of the eye including cornea, iris, lens, trabecular meshwork, and Schlemm canal. Anterior segment dysgeneses result from abnormal migration or differentiation of the neural crest derived mesenchymal cells that give rise to components of the anterior chamber during eye development. Different anterior segment anomalies may exist alone or in combination, including iris hypoplasia, enlarged or reduced corneal diameter, corneal vascularization and opacity, posterior embryotoxon, corectopia, polycoria, abnormal iridocorneal angle, ectopia lentis, and anterior synechiae between the iris and posterior corneal surface. Clinical conditions falling within the phenotypic spectrum of anterior segment dysgeneses include aniridia, Axenfeld anomaly, Reiger anomaly/syndrome, Peters anomaly, and iridogoniodysgenesis.

EXPRESSÃO TECIDUAL(Tecido-específico)
Músculo esquelético
9.3 TPM
Testículo
2.4 TPM
Substância negra
1.8 TPM
Coração - Ventrículo esquerdo
0.7 TPM
Rim - Medula
0.3 TPM
OUTRAS DOENÇAS (4)
cataract 11 multiple typesanterior segment dysgenesis 1early-onset posterior polar cataractcataract-glaucoma syndrome
HGNC:9006UniProt:O75364
FOXC1Forkhead box protein C1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

DNA-binding transcriptional factor that plays a role in a broad range of cellular and developmental processes such as eye, bones, cardiovascular, kidney and skin development (PubMed:11782474, PubMed:14506133, PubMed:14578375, PubMed:15277473, PubMed:15299087, PubMed:15684392, PubMed:16449236, PubMed:16492674, PubMed:17210863, PubMed:19279310, PubMed:19793056, PubMed:25786029, PubMed:27804176, PubMed:27907090). Acts either as a transcriptional activator or repressor (PubMed:11782474). Binds to th

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (2)
Formation of the ureteric budFormation of intermediate mesoderm
MECANISMO DE DOENÇA

Axenfeld-Rieger syndrome 3

An autosomal dominant disorder of morphogenesis that results in abnormal development of the anterior segment of the eye, and results in blindness from glaucoma in approximately 50% of affected individuals. Features include posterior corneal embryotoxon, prominent Schwalbe line and iris adhesion to the Schwalbe line, hypertelorism, hypodontia, sensorineural deafness, redundant periumbilical skin, and cardiovascular defects such as patent ductus arteriosus and atrial septal defect. When associated with tooth anomalies, the disorder is known as Rieger syndrome.

EXPRESSÃO TECIDUAL(Ubíquo)
Artéria tibial
171.9 TPM
Aorta
171.6 TPM
Artéria coronária
95.5 TPM
Glândula salivar
63.9 TPM
Nervo tibial
45.8 TPM
OUTRAS DOENÇAS (7)
Axenfeld-Rieger syndrome type 3anterior segment dysgenesis 3Rieger anomalyPeters anomaly
HGNC:3800UniProt:Q12948

Medicamentos aprovados (FDA)

1 medicamento encontrado nos registros da FDA americana.

💊 PredniSONE (PREDNISONE)
Ver no DailyMed/FDA

Variantes genéticas (ClinVar)

621 variantes patogênicas registradas no ClinVar.

🧬 CPAMD8: NM_015692.5(CPAMD8):c.3547G>C (p.Gly1183Arg) ()
🧬 CPAMD8: NM_015692.5(CPAMD8):c.4157C>A (p.Thr1386Asn) ()
🧬 CPAMD8: NM_015692.5(CPAMD8):c.2270del (p.Asn757fs) ()
🧬 CPAMD8: NM_015692.5(CPAMD8):c.1324C>T (p.Leu442Phe) ()
🧬 CPAMD8: NM_015692.5(CPAMD8):c.1758+71G>T ()
Ver todas no ClinVar

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Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

Dynamics of Anterior Chamber Angle Parameters in Primary and Secondary Pseudophakic Eyes Following Congenital Cataract Surgery.

Translational vision science & technology2026 Feb 02

To evaluate anterior chamber angle (ACA) parameter changes and their correlation with intraocular pressure (IOP) in primary versus secondary intraocular lens (IOL) implantation following congenital cataract (CC) surgery, using swept-source anterior segment optical coherence tomography (SS-ASOCT). Preoperative and postoperative (1 and 6 months) SS-ASOCT images from 89 eyes of 64 patients undergoing CC surgery were analyzed. ACA parameters (AOD500, ARA500, TISA500, TIA500) were measured. Generalized estimating equations assessed ACA parameter changes, and multivariable analysis determined relationships between ACA parameters and IOP. Compared to normal eyes, aphakic eyes showed larger AOD500 and ARA500 but smaller TISA500 (P < 0.05). After IOL implantation, both primary and secondary pseudophakic eyes (P-PsE and S-PsE) exhibited significant postoperative reductions in ARA500 and TISA500 (P < 0.05), mainly in the nasal angle. Multivariable analysis revealed corneal thickness positively correlated with IOP in both groups, while nasal AOD500 (β = -0.685, P < 0.05) and nasal TIA500 (β = -0.109; P < 0.001) showed negative associations in S-PsE. Angle abnormalities were identified during the annotation of images, associated with poorer visual outcomes. Significant postoperative reductions in ARA500 and TISA500 occurred in both primary and secondary IOL implantation for CC, predominantly in the nasal angle. Although overall angle parameters were comparable, S-PsEs uniquely demonstrated IOP correlations with pupil diameter and nasal angle, suggesting distinct IOP regulation mechanisms. SS-ASOCT-derived nasal anterior chamber angle metrics may provide an accessible biomarker for monitoring angle remodeling and intraocular pressure regulation following congenital cataract surgery.

#2

Congenital Corneal Staphyloma in an Asian Infant With Kabuki Syndrome Confirmed by a KMT2D Mutation.

The Journal of craniofacial surgery2026 Mar 24

This case report presents the first case of congenital corneal staphyloma in an Asian pediatric patient with Kabuki syndrome confirmed by a KMT2D gene mutation. A female infant, born at 38 weeks and 3 days of gestation via cesarean section, was referred immediately after birth for anterior segment dysgenesis of the right eye. The right eye showed microphthalmia accompanied by corneal opacity and limbal deficiency with neovascularization. Intraocular tumors were ruled out by ocular ultrasound and MRI. The left eye appeared normal. Subsequently, the cornea continued to protrude, leading to a clinical diagnosis of corneal staphyloma. At ∼7 months of age, she underwent enucleation with the insertion of an orbital hydroxyapatite implant. The postoperative pathology examination confirmed corneal staphyloma, identifying a fibrous stroma of squamous epithelium with pigmentation. Genetic testing identified a KMT2D gene mutation, confirming a diagnosis of Kabuki syndrome. Her left eye is developing normally with no structural abnormalities.

#3

Congenital lumbar and umbilical hernias in lumbo-costo-vertebral syndrome with ipsilateral renal agenesis: A rare case report with literature review.

International journal of surgery case reports2026 Mar

Congenital lumbar hernia (CLH) is a rare condition in infancy, often associated with lumbo-costo-vertebral syndrome (LCVS), which combines vertebral, rib, and abdominal wall anomalies. Genitourinary defects are uncommon and rarely documented. A 1-year-old girl presented with a left-sided CLH, small umbilical hernia, and prominent left anterior ribs protrusion manifesting as a chest wall bulge. Radiologic workup showed T10-T11 hemivertebrae, fused left laminae and ribs (10th-11th), scoliosis convex to the hernia side, and ipsilateral renal agenesis, confirming LCVS with a genitourinary anomaly. Elective open surgical repair included meshplasty of the left lumbar defect and excision of the protruding fused left anterior rib segment through the same incision, with concurrent umbilical hernia repair through a circumferential periumbilical incision. Recovery was uneventful. This case illustrates the syndromic nature of CLH within LCVS and underscores the need to screen for associated anomalies, particularly genitourinary defects such as renal agenesis. Imaging beyond ultrasound is often required to delineate skeletal and myofascial abnormalities. An open approach was favored to allow simultaneous excision of the protruding fused left rib segment and durable mesh reinforcement of a ~5 cm defect; this strategy achieved excellent outcomes. The coexistence of umbilical hernia with LCVS and CLH may be incidental but could broaden the recognized phenotypic spectrum and, to our knowledge, has not been previously reported. Awareness of LCVS in infants with CLH facilitates evaluation for associated anomalies and guides timely surgical management. Early recognition and repair can yield favorable results.

#4

Clinicopathologic Findings of Congenital Megalocornea in Three Blue-Throated Conures (Pyrrhura cruentata).

Veterinary ophthalmology2026 Mar

To describe the first known occurrence of megalocornea in a cohort of juvenile blue-throated conures (Pyrrhura cruentata). Three genetically related juvenile blue-throated conures. The information collected included physical and ophthalmic examination findings, ocular ultrasound biomicroscopy, Micro-CT analysis, postoperative outcomes following chemical cycloablation and/or enucleation, and histopathology. Ophthalmic examinations revealed multiple congenital ocular anomalies and bilateral blindness with the predominant feature of corneal enlargement. Several eyes developed corneal edema and exposure keratitis from lagophthalmos. Ultrasound biomicroscopy (UBM) showed a globoid cornea and enlarged anterior chamber with or without anterior displacement of the lens. Micro-CT analysis revealed normal skull conformation with bilateral enlargement of the anterior segments. An initial diagnosis of presumptive congenital glaucoma with buphthalmos was made. All conures had a chemical cycloablation procedure performed in the left eye to decrease ocular size. One conure had an enucleation of its right eye (OD) due to severe corneal ulceration. Two conures were humanely euthanized due to systemic health concerns shortly after initial presentation, and one died suddenly of a suspected cardiac event 8 years later. Ocular histopathology was performed on all 6 eyes of 3 conures, confirming a diagnosis of megalocornea with secondary ulcerative keratitis, stromal attenuation, and marked fibrosis with a discontinuous Descemet's membrane. This case series represents the first documented report of congenital megalocornea in blue-throated conures, which should be considered as a differential in birds with presumed buphthalmos from glaucoma.

#5

Bilateral Eyelid Agenesis With Multiple Congenital Ocular Anomalies in an Australian Labradoodle Puppy: Case Report and Surgical Management.

Veterinary ophthalmology2026 Mar

A 15-week-old Australian Labradoodle puppy was presented to The University of Queensland Small Animal Hospital for bilateral eyelid agenesis/coloboma and associated ocular complications. Enucleation of the left globe and repair of the right eyelid via a lip-to-lid transmucosal flap was performed. Given the subsequent development of prolapse of the gland of the nictitating membrane and a lack of vision of the right eye, enucleation of the right globe was then performed 2 months following the initial procedure. Both globes and a skin wedge from the right eye were submitted to The University of Queensland Veterinary Laboratory Service for histopathology assessment. Microscopic globe examination revealed lesions consistent with bilateral Peters' anomaly (anterior chamber cleavage syndrome, ACCS), characterized by axial defects in Descemet's membrane and the posterior corneal stroma, anterior synechiae at the site of the defects, and closure of the drainage angle. Other important histological findings were persistent pupillary membranes, retinal separation, and aphakia. Histopathology of the skin wedge from the right eye demonstrated complete absence of conjunctival goblet cells, tarsal plate, and meibomian glands, consistent with the finding of eyelid agenesis/coloboma. This report describes an uncommon and underreported presentation of bilateral canine lower eyelid agenesis with concurrent multiple congenital ocular anomalies, including bilateral Peters' anomaly.

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Current medical science
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A Case of Non-Syndromic Congenital Cataracts Caused by a Novel MAF Variant in the C-Terminal DNA-Binding Domain-Case Report and Literature Review.

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International immunopharmacology
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The neuroradiology journal
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In Silico Characterization of Pathogenic Homeodomain Missense Mutations in the PITX2 Gene.

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High Prevalence of Myocardial Bridging Detected in an Indonesian Population Using Multi-Detector Computed Tomography.

Medicina (Kaunas, Lithuania)
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Congenital Corneal Staphyloma: A Comprehensive Histopathologic Assessment Including Associated Anterior Segment Abnormalities.

Journal of pediatric ophthalmology and strabismus
2024

Stability between single and segmental maxillary osteotomies in bimaxillary surgery for cleft-associated class III deformity: a CBCT study.

Clinical oral investigations
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Acta ophthalmologica
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BMJ case reports
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Bilateral optic disc collaterals secondary to high-flow dural arteriovenous fistula: a diagnostic dilemma.

BMJ case reports
2024

Evaluating neural crest cell migration in a Col4a1 mutant mouse model of ocular anterior segment dysgenesis.

Cells &amp; development
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Spontaneous Coronary Artery Dissection in a Male Patient With Fibromuscular Dysplasia.

Texas Heart Institute journal
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International journal of ophthalmology
2024

Modernizing the evaluation of infantile nystagmus: the role of handheld optical coherence tomography.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2024

Macular and peripapillary vascular parameters in the fellow eyes of unilateral primary congenital glaucoma: a comparative study.

Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus
2024

Unusual Posterior Capsular Pigmentation in Axenfeld-Rieger Anomaly.

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BMJ case reports
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Transseptal coronary artery-a pictorial review.

Pediatric radiology
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Neurology
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Intergenic sequences harboring potential enhancer elements contribute to Axenfeld-Rieger syndrome by regulating PITX2.

JCI insight
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In Vivo Assessment of Retinal Phenotypes in Axenfeld-Rieger Syndrome.

Investigative ophthalmology &amp; visual science
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Retinal cases &amp; brief reports
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Seizure
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Primary Cilium in Neural Crest Cells Crucial for Anterior Segment Development and Corneal Avascularity.

Investigative ophthalmology &amp; visual science
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[Changes in tongue position and three-dimensional changes in upper airway before and after treatment with Twin-block combined with maxillary expansion appliance in children with mandibular retrusion].

Shanghai kou qiang yi xue = Shanghai journal of stomatology
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Structural Variant Disrupting the Expression of the Remote FOXC1 Gene in a Patient with Syndromic Complex Microphthalmia.

International journal of molecular sciences
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A bird's eye view on the use of whole exome sequencing in rare congenital ophthalmic diseases.

Journal of human genetics
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[Structural and functional features of the eye in Marfan syndrome. Report 2. Changes in the anatomical complex of the lens].

Vestnik oftalmologii
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[Structural and functional features of the eye in Marfan syndrome. Report 1. Changes in the fibrous tunic of the eye].

Vestnik oftalmologii
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Craniofacial bone anomalies related to cholesterol synthesis defects.

Scientific reports
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Myocardial bridging in obstructive hypertrophic cardiomyopathy: a risk factor for myocardial fibrosis.

BMC medicine
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Comparison and Evaluation of the Accuracy for Thoracic and Lumbar Pedicle Screw Fixation in Early-Onset Congenital Scoliosis Children.

Discovery medicine
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Neurotrophic Keratopathy in Marfan Syndrome Patient After Micropulse Transscleral Cyclophotocoagulation: A Call for Risk Stratification.

The American journal of case reports
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Ophthalmic &amp; physiological optics : the journal of the British College of Ophthalmic Opticians (Optometrists)
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Acta paediatrica (Oslo, Norway : 1992)
2024

Prenatal and Postnatal Ocular Abnormalities Following Congenital Zika Virus Infections: A Systematic Review.

Ocular immunology and inflammation
2024

[Liver failure caused by recurrent biliary bleeding associated with Osler's disease: a case report].

Nihon Shokakibyo Gakkai zasshi = The Japanese journal of gastro-enterology
2024

Comparative posterior corneal profile of keratoconus hydrops versus Haab's striae in congenital glaucoma.

Indian journal of ophthalmology
2024

Robotic-assisted single-site abdominal cerclage in the bicornuate uterus patient with cervical insufficiency.

Fertility and sterility
2024

Classifications of anterior segment structure of congenital corneal opacity in infants and toddlers by ultrasound biomicroscopy and slit-lamp microscopic photographs: an observational study.

BMC ophthalmology
2024

[Communication interventriculaire compliquant un infarctus de myocarde antérieur : un cas de fermeture percutanée].

Annales de cardiologie et d'angeiologie
2024

General Treatment and Ophthalmic Management of Peters' Anomaly.

Journal of clinical medicine
2024

Evaluation of transpalatal distraction in cleft palate patients.

Oral and maxillofacial surgery
2023

Genetics of the anterior segment dysgenesis.

Taiwan journal of ophthalmology
2023

New horizons in aniridia management: Clinical insights and therapeutic advances.

Taiwan journal of ophthalmology
2023

Congenital anomalies of lens shape.

Taiwan journal of ophthalmology
2023

Ocular Manifestations in Head and Neck Cancer: A Cross-Sectional Study from a Tertiary Care Centre from South India.

Romanian journal of ophthalmology
2024

Complex balanced intrachromosomal rearrangement involving PITX2 identified as a cause of Axenfeld-Rieger Syndrome.

American journal of medical genetics. Part A

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

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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. Dynamics of Anterior Chamber Angle Parameters in Primary and Secondary Pseudophakic Eyes Following Congenital Cataract Surgery.
    Translational vision science &amp; technology· 2026· PMID 41631743mais citado
  2. Congenital Corneal Staphyloma in an Asian Infant With Kabuki Syndrome Confirmed by a KMT2D Mutation.
    The Journal of craniofacial surgery· 2026· PMID 41875091mais citado
  3. Congenital lumbar and umbilical hernias in lumbo-costo-vertebral syndrome with ipsilateral renal agenesis: A rare case report with literature review.
    International journal of surgery case reports· 2026· PMID 41836995mais citado
  4. Clinicopathologic Findings of Congenital Megalocornea in Three Blue-Throated Conures (Pyrrhura cruentata).
    Veterinary ophthalmology· 2026· PMID 41807268mais citado
  5. Bilateral Eyelid Agenesis With Multiple Congenital Ocular Anomalies in an Australian Labradoodle Puppy: Case Report and Surgical Management.
    Veterinary ophthalmology· 2026· PMID 41766489mais citado
  6. Advancing therapies for anterior segment developmental anomalies.
    Taiwan J Ophthalmol· 2023· PMID 38249506recente
  7. Congenital corneal staphyloma in 8q21.11 microdeletion syndrome.
    Ophthalmic Genet· 2023· PMID 36341706recente
  8. Update on pediatric corneal diseases and keratoplasty.
    Surv Ophthalmol· 2022· PMID 35918016recente
  9. Genetic background modifies vulnerability to glaucoma-related phenotypes in Lmx1b mutant mice.
    Dis Model Mech· 2021· PMID 33462143recente
  10. The Oculome Panel Test: Next-Generation Sequencing to Diagnose a Diverse Range of Genetic Developmental Eye Disorders.
    Ophthalmology· 2019· PMID 30653986recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:88632(Orphanet)
  2. MONDO:0019503(MONDO)
  3. GARD:10025(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q3108583(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

Disgenesia mesenquimatosa do segmento anterior do olho, familiar
Compêndio · Raras BR

Disgenesia mesenquimatosa do segmento anterior do olho, familiar

ORPHA:88632 · MONDO:0019503
CID-11
Ensaios
1 ativos
Início
Neonatal
MedGen
UMLS
C1862839
Wikidata
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