Uma síndrome muito rara, caracterizada por cabelos ralos e curtos desde o nascimento, seguida de degeneração progressiva da mácula que leva à cegueira.
Introdução
O que você precisa saber de cara
Uma síndrome muito rara, caracterizada por cabelos ralos e curtos desde o nascimento, seguida de degeneração progressiva da mácula que leva à cegueira.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
Encontrou um erro ou informação desatualizada? Sugira uma correção →
Entender a doença
Do básico ao detalhe, leia no seu ritmo
Preparando trilha educativa...
Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 14 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 36 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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
1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.
Cadherins are calcium-dependent cell adhesion proteins. They preferentially interact with themselves in a homophilic manner in connecting cells; cadherins may thus contribute to the sorting of heterogeneous cell types
Cell membrane
Hypotrichosis congenital with juvenile macular dystrophy
A disorder characterized by congenital hypotrichosis, early hair loss, and severe degenerative changes of the retinal macula that culminate in blindness during the second to third decade of life.
Variantes genéticas (ClinVar)
118 variantes patogênicas registradas no ClinVar.
Vias biológicas (Reactome)
1 via biológica associada aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Hipotricose com degenerescência macular juvenil
Selecione um estado ou use sua localização para ver resultados.
Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.
Pesquisa ativa
Ensaios clínicos abertos e novidades científicas recentes
Pesquisa e ensaios clínicos
Nenhum ensaio clínico registrado para esta condição.
Publicações mais relevantes
Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.
Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.
Hypotrichosis with Juvenile Macular Dystrophy in a Patient with Cadherin 3 (CDH3) Mutation.
Hypotrichosis with juvenile macular dystrophy (HJMD) is an autosomal recessive disease with progressive macular degeneration leading to blindness in the first three decades of life along with hypotrichosis. We herein report a case of a five year old boy with hypotrichosis with juvenile macular dystrophy diagnosed with multi-modal imaging which was later confirmed by genetic testing by whole genome sequencing. Fundus examination of both eyes revealed symmetrical hypopigmentation in peripapillary retinal pigment epithelium (RPE) involving posterior pole and surrounded by a mottled hyperpigmented border. Fundus autofluorescence showed central hypo autofluorescence with surrounding hyper autofluorescence corresponding to RPE atrophy and a faint hypo autofluorescence at the junction of normal retina. SD-OCT showed segmental outer retinal and choriocapillaris atrophy temporal to fovea with interdigitation zone and ellipsoid zone loss and RPE irregularities with hyperreflective subretinal deposits at the fovea. Electroretinogram showed normal waves but a slight reduction of b wave amplitude in both eyes. He had sparse scalp-hair. Children with reduced vision not falling into a typical macular degeneration should be examined systemically and may just have sparse scalp hair and still have a genetic disease. A regular follow-up should be emphasized in view of progressive nature of the disease.
Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions.
Cadherins are a superfamily of calcium-dependent intercellular adhesion molecules that are widely expressed in living tissues. Within the retina and retinal pigment epithelium (RPE), cadherins contribute to tissue morphogenesis, neural circuit formation, adherens junctions of the outer blood-retinal barrier, photoreceptor disc morphogenesis, maintenance and survival. Four monogenic disorders involving genes which encode cadherins have been identified as causes of inherited retinal degeneration: the retinal cadherinopathies (CDHR1, CDH23, PCDH15, CDH3). Biallelic variants in CDHR1 result in cone-rod dystrophy, rod-cone dystrophy or late-onset macular dystrophy which may be misclassified as dry age-related macular degeneration. Biallelic variants in CDH23 and PCDH15 underlie Usher Syndrome type 1D and 1F. Hypotrichosis with juvenile macular dystrophy results from biallelic variants in CDH3, which contributes to adherens tight junctions between RPE cells. In this review, we summarise the classification of cadherins, and the role of cadherins in the physiology and morphogenesis of the inner and outer retina. Cadherins expressed in primate photoreceptors (CDHR1, CDH23 and PCDH15) have evolved complex roles in outer segment disc morphogenesis and maintenance involving intracellular heterophilic interactions which are as yet incompletely characterised. We highlight what is currently unknown about the molecular function of these cadherins, and review the pathogenesis, clinical phenotype and molecular genetics of each monogenic retinal cadherinopathy. Genes regulating the expression and post-translational modification of retinal cadherins, or those coding for as yet unidentified interacting partners, are candidates for unsolved cases of retinal degeneration. This group of disorders is potentially treatable; we summarise the likely molecular therapeutic approaches and future directions for each retinal cadherinopathy.
The first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.
Pathogenic variants in the Cadherin 3 (CDH3) gene are responsible for the occurrence of Hypotrichosis with Juvenile Macular Dystrophy (HJMD) and Ectodermal Dysplasia, Ectrodactyly and Macular Dystrophy Syndrome (EEMS), both of which are rare autosomal recessive disorders characterized by hypotrichosis and progressive macular dystrophy. The CDH3 gene encodes for P-cadherin, a calcium-binding protein that is essential for cell-cell adhesion, which is expressed in the retinal pigment epithelial cells and hair follicles. Fundus examination of both eyes was done in addition to clinical investigation. Genomic DNA was extracted from a whole-blood sample and whole-exome sequencing (WES) was performed to identify the underlying etiology.All identified variants were evaluated for their pathogenicity and causality. We present the first case of HJMD in a 23-year-old female patient from Jordan. The patient presented to our ophthalmology clinic with poor vision in both eyes. Gross examination revealed sparse scalp hair along with macular dystrophy on fundus exam in both eyes. HJMD was suspected and whole-exome sequencing (WES) confirmed the diagnosis with the identification of a homozygous frameshift deletion (p.Gly277AlafsTer20) localised in exon 7 of the CDH3 gene. Blindness due to progressive macular degeneration is a common manifestation in numerous syndromic recessive disorders such as HJMD. Ophthalmologists should consider the importance of systemic manifestations and genetic testing for the confirmation of diagnosis.
Publicações recentes
Hypotrichosis with Juvenile Macular Dystrophy in 2 Brothers.
The First Colombian Patient with CDH3-Related Hypotrichosis with Juvenile Macular Dystrophy.
Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.
Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.
📚 EuropePMC1 artigos no totalmostrando 22
Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.
The Australasian journal of dermatologyClinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.
Clinical dysmorphologyHypotrichosis with Juvenile Macular Dystrophy in a Patient with Cadherin 3 (CDH3) Mutation.
Nepalese journal of ophthalmology : a biannual peer-reviewed academic journal of the Nepal Ophthalmic Society : NEPJOPHNovel compound heterozygous cadherin 3 mutations in hypotrichosis and juvenile macular dystrophy.
Chinese medical journalRetinal cadherins and the retinal cadherinopathies: Current concepts and future directions.
Progress in retinal and eye researchThe first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.
Ophthalmic geneticsMultimodal imaging of Hypotrichosis with juvenile macular dystrophy: a case report.
BMC ophthalmologyMultimodal Imaging in Hypotrichosis with Juvenile Macular Degeneration.
Ophthalmology. RetinaThe first Japanese family of CDH3-related hypotrichosis with juvenile macular dystrophy.
Molecular genetics & genomic medicineCorrelating Adaptive Optics Images to Clinical Findings in Juvenile Macular Dystrophy with Hypotrichosis in Siblings with Homozygous CDH3 Pathogenic Variation.
Ophthalmic researchHypotrichosis with juvenile macular dystrophy.
Ophthalmic geneticsNovel CDH3 variants in Brazilian families with hypotrichosis and juvenile macular dystrophy revealed by exome sequencing.
Clinical geneticsHypotrichosis with juvenile macular dystrophy: Combination of whole-genome sequencing and genome-wide homozygosity mapping identifies a large deletion in CDH3 initially undetected by whole-exome sequencing-A lesson from next-generation sequencing.
Molecular genetics & genomic medicineHypotrichosis with cone-rod dystrophy in a patient with cadherin 3 (CDH3) mutation.
Documenta ophthalmologica. Advances in ophthalmologyHypotrichosis with juvenile macular dystrophy: a case report with molecular study.
Arquivos brasileiros de oftalmologiaNew CDH3 mutation in the first Spanish case of hypotrichosis with juvenile macular dystrophy, a case report.
BMC medical geneticsHypotrichosis with juvenile macular dystrophy: Portuguese case.
Dermatology online journalNovel homozygous sequence variants in the CDH3 gene encoding P-cadherin underlying hypotrichosis with juvenile macular dystrophy in consanguineous families.
European journal of dermatology : EJDCharacterization of CDH3-Related Congenital Hypotrichosis With Juvenile Macular Dystrophy.
JAMA ophthalmologyHypotrichosis and juvenile macular dystrophy caused by CDH3 mutation: A candidate disease for retinal gene therapy.
Scientific reportsPhenotypic observations in "hypotrichosis with juvenile macular dystrophy" (recessive CDH3 mutations).
Ophthalmic geneticsA case of hypotrichosis with juvenile macular dystrophy.
Retinal cases & brief reportsAssociações
Organizações que acompanham esta doença — pra ter apoio e orientação
Ainda não temos associações cadastradas para Hipotricose com degenerescência macular juvenil.
É de uma associação que acompanha esta doença? Fale com a gente →
Comunidades
Grupos ativos de quem convive com esta doença aqui no Raras
Ainda não existe comunidade no Raras para Hipotricose com degenerescência macular juvenil
Pacientes, familiares e cuidadores se organizam em comunidades pra compartilhar experiências, fazer perguntas e se apoiar. Você pode ser o primeiro.
Tire suas dúvidas
Perguntas, dicas e experiências compartilhadas aqui na página
Participe da discussão
Faça login para postar dúvidas, compartilhar experiências e interagir com especialistas.
Fazer loginDoenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
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.
- Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.
- Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.
- Hypotrichosis with Juvenile Macular Dystrophy in a Patient with Cadherin 3 (CDH3) Mutation.Nepalese journal of ophthalmology : a biannual peer-reviewed academic journal of the Nepal Ophthalmic Society : NEPJOPH· 2022· PMID 35996915mais citado
- Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions.
- The first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.
- Hypotrichosis with Juvenile Macular Dystrophy in 2 Brothers.
- The First Colombian Patient with CDH3-Related Hypotrichosis with Juvenile Macular Dystrophy.
- Correction: Multimodal imaging of Hypotrichosis with juvenile macular dystrophy: a case report.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:1573(Orphanet)
- OMIM OMIM:601553(OMIM)
- MONDO:0011107(MONDO)
- GARD:3066(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q22132220(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
