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Hipotricose com degenerescência macular juvenil
ORPHA:1573CID-10 · Q84.0CID-11 · LD27.0YOMIM 601553DOENÇA RARA

Uma síndrome muito rara, caracterizada por cabelos ralos e curtos desde o nascimento, seguida de degeneração progressiva da mácula que leva à cegueira.

Mantido por Agente Raras·Colaborar como especialista →

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.

Publicações científicas
46 artigos
Último publicado: 2025 Oct 21

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
<1 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Casos conhecidos
50
pacientes catalogados
Início
Infancy
+ neonatal
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q84.0
🇧🇷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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Entender a doença

Do básico ao detalhe, leia no seu ritmo

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Sinais e sintomas

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

Partes do corpo afetadas

👁️
Olhos
9 sintomas
🧬
Pele e cabelo
8 sintomas
🦴
Ossos e articulações
3 sintomas
🛡️
Imunológico
1 sintomas
🦷
Dentes
1 sintomas

+ 14 sintomas em outras categorias

Características mais comuns

100%prev.
Pili torti
Frequente (79-30%)
100%prev.
Degeneração macular
Muito frequente (99-80%)
100%prev.
Cabelo esparso no couro cabeludo
Muito frequente (99-80%)
100%prev.
Palidez
Obrigatório (100%)
100%prev.
Glândulas sebáceas ausentes
Obrigatório (100%)
100%prev.
Distrofia macular
Obrigatório (100%)
36sintomas
Muito frequente (21)
Frequente (2)
Ocasional (7)
Muito raro (5)
Sem dados (1)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 36 características clínicas mais associadas, ordenadas por frequência.

Pili torti
Frequente (79-30%)100%
Degeneração macularMacular degeneration
Muito frequente (99-80%)100%
Cabelo esparso no couro cabeludoSparse scalp hair
Muito frequente (99-80%)100%
PalidezPallor
Obrigatório (100%)100%
Glândulas sebáceas ausentesAbsent sebaceous glands
Obrigatório (100%)100%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa2desde 2024
Total histórico46PubMed
Últimos 10 anos23publicações
Pico20165 papers
Linha do tempo
2024Hoje · 2026📈 2016Ano 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

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.

CDH3Cadherin-3Disease-causing germline mutation(s) inTolerante
FUNÇÃO

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

LOCALIZAÇÃO

Cell membrane

VIAS BIOLÓGICAS (1)
Adherens junctions interactions
MECANISMO DE DOENÇA

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.

OUTRAS DOENÇAS (2)
congenital hypotrichosis with juvenile macular dystrophyEEM syndrome
HGNC:1762UniProt:P22223

Variantes genéticas (ClinVar)

118 variantes patogênicas registradas no ClinVar.

🧬 CDH3: NM_001793.6(CDH3):c.1677_1710del (p.Ser559fs) ()
🧬 CDH3: NM_001793.6(CDH3):c.119_122del (p.Ala40fs) ()
🧬 CDH3: NM_001793.6(CDH3):c.2133+54C>T ()
🧬 CDH3: NM_001793.6(CDH3):c.1182+45C>G ()
🧬 CDH3: NM_001793.6(CDH3):c.46-44C>T ()
Ver todas no ClinVar

Vias biológicas (Reactome)

1 via biológica associada aos genes desta condição.

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Carregando informações de tratamento...

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.

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Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.

The Australasian journal of dermatology2024 Feb
#2

Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.

Clinical dysmorphology2023 Apr 01
#3

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 : NEPJOPH2022 Jan

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.

#4

Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions.

Progress in retinal and eye research2022 Sep

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.

#5

The first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.

Ophthalmic genetics2022 Jun

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

Ver todas no PubMed

📚 EuropePMC1 artigos no totalmostrando 22

2024

Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.

The Australasian journal of dermatology
2023

Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.

Clinical dysmorphology
2022

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

Novel compound heterozygous cadherin 3 mutations in hypotrichosis and juvenile macular dystrophy.

Chinese medical journal
2022

Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions.

Progress in retinal and eye research
2022

The first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.

Ophthalmic genetics
2021

Multimodal imaging of Hypotrichosis with juvenile macular dystrophy: a case report.

BMC ophthalmology
2021

Multimodal Imaging in Hypotrichosis with Juvenile Macular Degeneration.

Ophthalmology. Retina
2021

The first Japanese family of CDH3-related hypotrichosis with juvenile macular dystrophy.

Molecular genetics &amp; genomic medicine
2020

Correlating Adaptive Optics Images to Clinical Findings in Juvenile Macular Dystrophy with Hypotrichosis in Siblings with Homozygous CDH3 Pathogenic Variation.

Ophthalmic research
2019

Hypotrichosis with juvenile macular dystrophy.

Ophthalmic genetics
2020

Novel CDH3 variants in Brazilian families with hypotrichosis and juvenile macular dystrophy revealed by exome sequencing.

Clinical genetics
2019

Hypotrichosis 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 &amp; genomic medicine
2019

Hypotrichosis with cone-rod dystrophy in a patient with cadherin 3 (CDH3) mutation.

Documenta ophthalmologica. Advances in ophthalmology
2017

Hypotrichosis with juvenile macular dystrophy: a case report with molecular study.

Arquivos brasileiros de oftalmologia
2017

New CDH3 mutation in the first Spanish case of hypotrichosis with juvenile macular dystrophy, a case report.

BMC medical genetics
2016

Hypotrichosis with juvenile macular dystrophy: Portuguese case.

Dermatology online journal
2016

Novel homozygous sequence variants in the CDH3 gene encoding P-cadherin underlying hypotrichosis with juvenile macular dystrophy in consanguineous families.

European journal of dermatology : EJD
2016

Characterization of CDH3-Related Congenital Hypotrichosis With Juvenile Macular Dystrophy.

JAMA ophthalmology
2016

Hypotrichosis and juvenile macular dystrophy caused by CDH3 mutation: A candidate disease for retinal gene therapy.

Scientific reports
2016

Phenotypic observations in "hypotrichosis with juvenile macular dystrophy" (recessive CDH3 mutations).

Ophthalmic genetics
2015

A case of hypotrichosis with juvenile macular dystrophy.

Retinal cases &amp; brief reports

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

Ainda não existe comunidade no Raras para Hipotricose com degenerescência macular juvenil

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Doenças 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.

  1. Hypotrichosis with juvenile macular dystrophy with novel mutations in CDH3 gene.
    The Australasian journal of dermatology· 2024· PMID 37850495mais citado
  2. Clinical, genetic, and electron microscopy of hair findings in a patient with CDH3 -related hypotrichosis with juvenile macular dystrophy.
    Clinical dysmorphology· 2023· PMID 36779776mais citado
  3. 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
  4. Retinal cadherins and the retinal cadherinopathies: Current concepts and future directions.
    Progress in retinal and eye research· 2022· PMID 35066146mais citado
  5. The first reported case of CDH3-related hypotrichosis with juvenile macular dystrophy from Jordan: a case report.
    Ophthalmic genetics· 2022· PMID 35038959mais citado
  6. Hypotrichosis with Juvenile Macular Dystrophy in 2 Brothers.
    Ophthalmol Retina· 2025· PMID 41117728recente
  7. The First Colombian Patient with CDH3-Related Hypotrichosis with Juvenile Macular Dystrophy.
    Skin Appendage Disord· 2025· PMID 40330852recente
  8. Correction: Multimodal imaging of Hypotrichosis with juvenile macular dystrophy: a case report.
    BMC Ophthalmol· 2024· PMID 38243158recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:1573(Orphanet)
  2. OMIM OMIM:601553(OMIM)
  3. MONDO:0011107(MONDO)
  4. GARD:3066(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. 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

Hipotricose com degenerescência macular juvenil
Compêndio · Raras BR

Hipotricose com degenerescência macular juvenil

ORPHA:1573 · MONDO:0011107
Prevalência
<1 / 1 000 000
Casos
50 casos conhecidos
Herança
Autosomal recessive
CID-10
Q84.0 · Alopécia congênita
CID-11
Início
Infancy, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C1832162
Repurposing
3 candidatos
anecortave-acetateangiogenesis inhibitor
luteinantioxidant
verteporfinphotosensitizing agent
EuropePMC
Wikidata
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