Uma doença de pele genética e de nascença, que afeta a pele e as mucosas (revestimentos internos). Ela se caracteriza por uma pele muito esticada e fina, com feridas e descamação, e está associada a características típicas no rosto, contraturas múltiplas nas articulações, uma sequência de deformidades causada pela falta ou redução dos movimentos do feto (bebê) ainda no útero (conhecida como FADS), e pulmões subdesenvolvidos, sem problemas neurológicos.
Introdução
O que você precisa saber de cara
A dermopatia restritiva é uma doença genética e congênita extremamente rara que afeta a formação da pele e das articulações do bebê ainda dentro do útero. Ao nascer, o bebê apresenta a pele muito fina, rígida e esticada, associada a articulações endurecidas e graves dificuldades para respirar devido a pulmões pouco desenvolvidos. A condição não tem cura nem conta com remédios específicos aprovados, e os cuidados hospitalares são voltados a dar suporte e conforto para a criança. No Brasil, a doença não consta na lista oficial de PCDTs do Ministério da Saúde, mas o SUS oferece exames diagnósticos como cariótipo e sequenciamento do exoma para a investigação familiar.
Uma doença de pele genética e de nascença, que afeta a pele e as mucosas (revestimentos internos). Ela se caracteriza por uma pele muito esticada e fina, com feridas e descamação, e está associada a características típicas no rosto, contraturas múltiplas nas articulações, uma sequência de deformidades causada pela falta ou redução dos movimentos do feto (bebê) ainda no útero (conhecida como FADS), e pulmões subdesenvolvidos, sem problemas neurológicos.
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
+ 40 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 95 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
2 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant, Autosomal recessive.
Emery-Dreifuss muscular dystrophy 2, autosomal dominant
A form of Emery-Dreifuss muscular dystrophy, a degenerative myopathy characterized by weakness and atrophy of muscle without involvement of the nervous system, early contractures of the elbows, Achilles tendons and spine, and cardiomyopathy associated with cardiac conduction defects.
Mandibuloacral dysplasia with type B lipodystrophy
A form of mandibuloacral dysplasia, a rare progeroid disorder with clinical and genetic heterogeneity, characterized by growth retardation, craniofacial dysmorphic features due to distal bone resorption, musculoskeletal and skin abnormalities associated with lipodystrophy. MADB is a disease characterized by mandibular and clavicular hypoplasia, acroosteolysis, delayed closure of the cranial suture, joint contractures, and generalized lipodystrophy with loss of subcutaneous fat from the extremities, face, neck and trunk.
Variantes genéticas (ClinVar)
967 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 195 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
8 vias biológicas associadas 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 — Dermopatia restritiva
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
Restrictive Dermopathy in Neonates.
L-Carnitine in Dermatology: A Systematic Review of Therapeutic Potential and Biomarker Applications.
L-Carnitine, an amino acid derivative critical for fatty acid metabolism, exhibits anti-inflammatory, antioxidant, and cytoprotective properties, with emerging applications in dermatology. This review explores its therapeutic potential, both topically and systemically, and its role as a biomarker in various skin disorders. A systematic search of Medline, EMBASE, and PubMed (1946-January 2025) was conducted, focusing on L-carnitine and dermatologic conditions, excluding acetyltransferases, acyltransferases, breast, and genetic metabolic diseases. From 420 articles, 59 were included after screening in Covidence. Data on study design, sample size, carnitine type, and outcomes were extracted and validated by two authors. Topically, L-carnitine (1%-5%) reduces acne lesions, sebum production, post-inflammatory hyperpigmentation, and cellulite, particularly when combined with agents like licochalcone-A or salicylic acid. Systemic L-carnitine mitigates inflammation, oxidative stress, and treatment-related side effects (eg, from corticosteroids, isotretinoin, vismodegib) while improving skin elasticity, microcirculation, wound healing, and fibrosis in conditions like Raynaud's disease, venous leg ulcers, and sclerotic disorders. As a biomarker, altered carnitine levels and metabolites reflect disease mechanisms in genodermatoses (eg, restrictive dermopathy, recessive dystrophic epidermolysis bullosa), inflammatory dermatoses, and melanoma, aiding diagnosis and monitoring. L-Carnitine shows promise as a therapeutic and diagnostic tool in dermatology, with benefits in acne, sebum control, post-inflammatory hyperpigmentation, cellulite, inflammation, and tissue repair. Its biomarker potential enhances disease insights and personalized care. Further dermatology-specific trials are needed to optimize dosing, formulations, and long-term safety, particularly regarding trimethylamine-N-oxide-related cardiovascular risks.
A Case of Restrictive Dermopathy With Atypical Cardiac Anomalies and a Novel ZMPSTE24 Variant.
Restrictive Dermopathy (RD, OMIM #275210) is an ultra-rare, lethal genodermatosis caused by defects in lamins and related proteins. RD is caused by biallelic pathogenic variants in ZMPSTE24, which encodes zinc metallopeptidase ZMPSTE24, an enzyme essential for processing prelamin A, the precursor of lamin A. While null variants leading to a total loss of prelamin A processing have been related to neonatally lethal RD, variants preserving residual prelamin A processing function cause an allelic disorder called Mandibuloacral Dysplasia Type B (MAD-B). RD is characterized by taut translucent skin, visible superficial vessels, joint contractures, and dysmorphic features, with death usually occurring within the first month of life. Cardiac anomalies, including ASD and PDA, have been reported in a few patients, most of whom were not genetically confirmed, and transposition of the great arteries (TGA) has been described only once, also without molecular confirmation. We report a patient with restrictive dermatopathy (RD) presenting with double outlet right ventricle (DORV) and pulmonary valve atresia which have not been previously reported in association with RD. Exome Sequencing (ES) was performed, revealing a novel homozygous splice-site variant in ZMPSTE24 (c.1203 + 1G>T). Segregation analysis was performed in the mother and two siblings. To our knowledge, DORV has not previously been reported in an RD patient. This case expands the genotypic spectrum of RD and suggests a possible link with complex cardiac malformations.
P18 ZMPSTE24 variant with the lethal phenotype of restrictive dermopathy.
Restrictive dermopathy (RD) is a rare, lethal, congenital autosomal recessive laminopathy syndrome characterized by generalised tight translucent skin, dysmorphic facies, arthrogryposis multiplex congenita, and pulmonary hypoplasia. It is caused by mutations in either ZMPSTE24 or LMNA. Variants in these genes, which disrupt the production of lamin A, and hence the structural integrity of the nuclear envelope, can also cause survivable syndromes such as mandibuloacral dysplasia (MAD). We report the first son of non-related Caucasian parents delivered via emergency LSCS at 34 weeks following preeclampsia, gestational diabetes and polyhydramnios. Extraction was difficult due to arthrogryposis. At birth (weight 1.65 kg), he showed the typical features of RD. Genetic testing revealed a homozygous pathogenic ZMPSTE24 frameshift variant c.1085dup p. (Leu362PhefsTer19) previously reported in mandibuloacral dysplasia (Agarwal AK, Fryns JP, Auchus RJ, Garg A. Zinc metalloproteinase, ZMPSTE24, is mutated in mandibuloacral dysplasia. Hum Mol Genet 2003;12:1995-2001). Parents were heterozygous carriers. The baby received palliative care and died on day 2. The longest survival in RD is 120 days, in contrast to other laminopathies. Similar or identical mutation within the LMNA gene produces varied phenotypes which expands the spectrum. Currently, there is no curative therapy for RD. Our main aim to diagnose this condition is to offer counselling, genetic testing for future pregnancies, prevent unnecessary interventions and support the family through compassion and dignity.
A case of restrictive dermopathy in a Hutterite newborn: Diagnosis and creative skin-directed management.
Restrictive dermopathy is a lethal autosomal recessive disease characterized by tightly adherent skin, distinctive facial dysmorphisms, arthrogryposis, and pulmonary hypoplasia. While clinical findings are unique, histopathology and genetic analysis are critical for early diagnostic confirmation and to initiate appropriate management for this lethal disease. We report on a preterm Hutterite male neonate with biallelic ZMPSTE24 mutations to highlight the clinical and histopathological features of restrictive dermopathy and share our skin-directed management strategies.
Publicações recentes
L-Carnitine in Dermatology: A Systematic Review of Therapeutic Potential and Biomarker Applications.
A Case of Restrictive Dermopathy With Atypical Cardiac Anomalies and a Novel ZMPSTE24 Variant.
Restrictive Dermopathy in Neonates.
P18 ZMPSTE24 variant with the lethal phenotype of restrictive dermopathy.
A case of restrictive dermopathy in a Hutterite newborn: Diagnosis and creative skin-directed management.
📚 EuropePMC83 artigos no totalmostrando 37
L-Carnitine in Dermatology: A Systematic Review of Therapeutic Potential and Biomarker Applications.
Journal of cutaneous medicine and surgeryA Case of Restrictive Dermopathy With Atypical Cardiac Anomalies and a Novel ZMPSTE24 Variant.
American journal of medical genetics. Part ARestrictive Dermopathy in Neonates.
Indian journal of pediatricsP18 ZMPSTE24 variant with the lethal phenotype of restrictive dermopathy.
The British journal of dermatologyA case of restrictive dermopathy in a Hutterite newborn: Diagnosis and creative skin-directed management.
Pediatric dermatologyRestrictive Dermopathy Due to ZMPSTE24 Mutation: A Case Report with a Novel Finding of Corpus Callosum Agenesis.
Indian journal of dermatologyPrelamin A and ZMPSTE24 in premature and physiological aging.
Nucleus (Austin, Tex.)PHGDH-related microcephalic dwarfism in two fetuses: Expanding the phenotypical spectrum of L-serine biosynthesis defect.
European journal of medical geneticsAnalysis of a non-lethal biallelic frameshift mutation in ZMPSTE24 reveals utilization of alternative translation initiation codons.
Clinical geneticsRestrictive dermopathy due to ZMPSTE24 deficiency.
Clinical dysmorphologyRestrictive dermopathy: A baby with taut skin, facial dysmorphism, joint contractures, and pulmonary hypoplasia.
JAAD case reportsLamin A to Z in normal aging.
AgingPrenatal Diagnosis of Neu-Laxova Syndrome.
Diagnostics (Basel, Switzerland)An exceptional biallelic N-terminal frame shift mutation in ZMPSTE24 leads to non-lethal progeria due to possible utilization of a downstream alternative start codon.
GeneNovel compound heterozygous indel ZMPSTE24 mutations in a Taiwanese male infant with restrictive dermopathy.
The Journal of dermatologyLethal Restrictive Dermopathy with ZMPSTE24 Mutation.
Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology SocietyRestrictive dermopathy: Three new patients with ZMPSTE24 mutations and a review of the literature.
Pediatric dermatologyAdenine base editing to treat progeria syndrome and extend the lifespan.
The journal of cardiovascular agingMutations Involved in Premature-Ageing Syndromes.
The application of clinical geneticsRestrictive Dermopathy - A Rare Congenital Skin Disorder.
Indian journal of dermatologyNeonatal presentation of COG6-CDG with prominent skin phenotype.
JIMD reportsNovel and recurrent PHGDH and PSAT1 mutations in Chinese patients with Neu-Laxova syndrome.
European journal of dermatology : EJDTeledermatology diagnosis of the first Italian patient affected with restrictive dermopathy due to ZMPSTE24 homozygous mutation.
Journal of the European Academy of Dermatology and Venereology : JEADVLamins and bone disorders: current understanding and perspectives.
OncotargetZMPSTE24 missense mutations that cause progeroid diseases decrease prelamin A cleavage activity and/or protein stability.
Disease models & mechanismsRestrictive Dermopathy: Four Case Reports and Structural Skin Changes.
Acta dermato-venereologicaPhenotypic heterogeneity of ZMPSTE24 deficiency.
American journal of medical genetics. Part AUnderstanding fetal factors that contribute to preterm birth: Sjögren-Larsson syndrome as a model.
Journal of perinatal medicineLethal multiple pterygium syndrome: A severe phenotype associated with a novel mutation in the nebulin gene.
Neuromuscular disorders : NMDProgeroid syndrome patients with ZMPSTE24 deficiency could benefit when treated with rapamycin and dimethylsulfoxide.
Cold Spring Harbor molecular case studiesRestrictive Dermopathy: A Rare Disease with Unusual Radiographic Findings.
Delaware medical journalDevelopment of a diagnostic DNA chip to screen for 30 autosomal recessive disorders in the Hutterite population.
Molecular genetics & genomic medicineA Heterozygous ZMPSTE24 Mutation Associated with Severe Metabolic Syndrome, Ectopic Fat Accumulation, and Dilated Cardiomyopathy.
CellsFrame shift mutations of the ZMPSTE24 gene in two siblings with restrictive dermopathy.
Clinical dysmorphologySkin Disease in Laminopathy-Associated Premature Aging.
The Journal of investigative dermatologyDNA repair defects and genome instability in Hutchinson-Gilford Progeria Syndrome.
Current opinion in cell biologySustained accumulation of prelamin A and depletion of lamin A/C both cause oxidative stress and mitochondrial dysfunction but induce different cell fates.
Nucleus (Austin, Tex.)Associações
Organizações que acompanham esta doença — pra ter apoio e orientação
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Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Dermopatia restritiva e no aconselhamento genético da família.
Doenças relacionadas
Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico
Perguntas frequentes
O que as famílias mais perguntam sobre esta doença — cada resposta com a fonte de onde saiu
A dermopatia restritiva é uma doença genética rara, congênita, caracterizada por pele muito fina e esticada, associada a contraturas nas articulações e pulmões subdesenvolvidos. Ela se manifesta desde a fase fetal e no período neonatal imediato.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
5 publicações do grafo RarasNet (PubMed) · 6 bases de dados. Títulos, periódicos e PMIDs vêm direto da fonte, sem intermediação de IA.
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.
Citar este verbete
Raras. (s.d.). Dermopatia restritiva. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/dermopatia-restritiva
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
Dermopatia restritiva
📋 Origem dos dados
Esta página agrega dados de fontes públicas e oficiais. Dados sobre cobertura no SUS (PCDT, CEAF) são verificados ativamente por agente proativo (ver badge no infobox). Demais dados têm atribuição de fonte + data da última sincronização — clique para abrir o original.
- Doença rara (ontologia)
- fonte: Orphanet
- Identificador unificado
- fonte: MONDO
- Codificação WHO/SUS
- fonte: WHO ICD-10 / DATASUS
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata