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Hipoplasia cartilagem-cabelo
ORPHA:175CID-10 · Q78.8CID-11 · LD27.0YOMIM 250250DOENÇA RARA

A hipoplasia cartilagem-cabelo é uma doença que afeta as extremidades dos ossos onde eles crescem, e causa baixa estatura desde o nascimento.

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Introdução

O que você precisa saber de cara

📋

A hipoplasia cartilagem-cabelo é uma doença que afeta as extremidades dos ossos onde eles crescem, e causa baixa estatura desde o nascimento.

Publicações científicas
281 artigos
Último publicado: 2026 Apr

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
150.0
Specific population
Início
Antenatal
+ infancy, neonatal
🏥
SUS: Cobertura mínimaScore: 15%
CID-10: Q78.8
🇧🇷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

Preparando trilha educativa...

Sinais e sintomas

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

Partes do corpo afetadas

🦴
Ossos e articulações
31 sintomas
😀
Face
9 sintomas
🩸
Sangue
7 sintomas
👁️
Olhos
7 sintomas
🫃
Digestivo
6 sintomas
🧬
Pele e cabelo
5 sintomas

+ 44 sintomas em outras categorias

Características mais comuns

96%prev.
Hipermobilidade articular
Ocasional (29-5%)
93%prev.
Cabelo claro
Frequência: 81/87
93%prev.
Cabelo esparso
Muito frequente (99-80%)
92%prev.
Extensão limitada do cotovelo
Muito frequente (99-80%)
90%prev.
Anormalidade no EEG
Muito frequente (99-80%)
90%prev.
Displasia metafisária
Muito frequente (99-80%)
123sintomas
Muito frequente (45)
Frequente (17)
Ocasional (29)
Muito raro (3)
Sem dados (29)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 123 características clínicas mais associadas, ordenadas por frequência.

Hipermobilidade articularJoint hypermobility
Ocasional (29-5%)96%
Cabelo claroFair hair
Frequência: 81/8793%
Cabelo esparsoSparse hair
Muito frequente (99-80%)93%
Extensão limitada do cotoveloLimited elbow extension
Muito frequente (99-80%)92%
Anormalidade no EEGEEG abnormality
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico281PubMed
Últimos 10 anos90publicações
Pico201713 papers
Linha do tempo
2026Hoje · 2026🧪 1998Primeiro ensaio clínico📈 2017Ano 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.

RMRPDisease-causing germline mutation(s) inDesconhecido
LOCALIZAÇÃO

OUTRAS DOENÇAS (5)
metaphyseal dysplasia without hypotrichosisanauxetic dysplasia 1cartilage-hair hypoplasiaOmenn syndrome
HGNC:10031

Variantes genéticas (ClinVar)

418 variantes patogênicas registradas no ClinVar.

🧬 RMRP: NC_000009.12:g.35658038_35658039insACAGCTTCACAGAG ()
🧬 RMRP: GRCh38/hg38 9p24.3-q21.13(chr9:208455-72054336)x3 ()
🧬 RMRP: GRCh38/hg38 9p24.3-13.1(chr9:208455-38787483)x3 ()
🧬 RMRP: NC_000009.12:g.35658017_35658043dup ()
🧬 RMRP: NC_000009.12:g.35658022_35658023insTCTCAGCTTCACAGA ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 3 variantes classificadas pelo ClinVar.

2
1
Patogênica (66.7%)
VUS (33.3%)
VARIANTES MAIS SIGNIFICATIVAS
RMRP: NR_003051.4(RMRP):n.213C>G [Likely pathogenic]
RMRP: NC_000009.12:g.35657872C>T [Likely pathogenic]
RMRP: NR_003051.4(RMRP):n.2G>A [Uncertain significance]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
Aprovado1
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 1 ensaio
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Hipoplasia cartilagem-cabelo

🗺️

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

2 ensaios clínicos encontrados.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

📖Melhor nível de evidência: Revisão
Timeline de publicações
92 papers (10 anos)
#1

Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.

BMJ case reports2026 Feb 20

Cartilage-hair hypoplasia (CHH) is a rare autosomal recessive skeletal dysplasia. It is often associated with short stature, metaphyseal abnormalities, hair hypoplasia and immune dysfunction. This case describes a pregnant woman in her mid-30s with two previous children diagnosed with CHH with molecular confirmation of two pathogenic variants of the ribonuclease mitochondrial RNA processing gene. The couple is healthy and non-consanguineous. Routine ultrasound examination of the current pregnancy suggested this fetus was also likely to be affected. The couple opted not to perform invasive prenatal diagnosis. Umbilical cord blood was collected at birth for genetic testing, confirming the diagnosis. CHH has a significant impact on the quality of life of those affected. Genetic counselling may help parents understand the disease and its prognosis. Obstetric ultrasound plays an important role in the diagnosis by allowing early detection of suspected cases as well as assessing fetal growth.

#2

Neonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.

European journal of medical genetics2026 Mar

Cartilage hair hypoplasia (CHH) syndrome (OMIM #250250) is a rare autosomal recessive metaphyseal dysplasia, characterized by disproportionate short stature, hypotrichosis and variable extra-skeletal manifestations, including immunodeficiency, anemia, intestinal diseases, and predisposition to malignancies. CHH results from homozygous or compound heterozygous mutations in the RMRP gene on chromosome 9p13, which encodes an untranslated RNA component of mitochondrial RNA-processing endoribonuclease. RMRP pathogenic variants can also lead to Omenn Syndrome (OS) (OMIM #603554), a systemic inflammatory condition displaying neonatal erythroderma and immunodeficiency. This report highlights the genotypic and phenotypic overlap between CHH and OS, by presenting a newborn with skeletal dysplasia, immunodeficiency and neonatal onset erythroderma, carrying the homozygous NR_003051:n.35C > A variant in the RMRP gene.

#3

Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.

Acta medica portuguesa2026 Jan 12

Cartilage-hair hypoplasia is a rare autosomal recessive skeletal dysplasia. It is particularly prevalent in the Finnish and Amish populations but increasing reports have been documented worldwide. It is caused by pathogenic variants in the RMRP gene. The clinical presentation is highly variable and may include short-limbed short stature, metaphyseal abnormalities, hypotrichosis, and immune deficiency, among other features. Some of the manifestations may present early in the prenatal period and ultrasound assessment is often the tool that raises suspicion for this condition. This review aims to summarize the current knowledge regarding the prenatal diagnosis of cartilage-hair hypoplasia, focusing on its molecular basis and the role of imaging and genetic testing. A comprehensive literature search was conducted in the PubMed/MEDLINE database using the terms 'Prenatal diagnosis', 'Cartilage-hair hypoplasia', 'Skeletal dysplasias', 'Osteochondrodysplasias' and 'RMRP mutation'. Prenatal diagnosis of this condition remains challenging, as ultrasound findings may overlap with other skeletal dysplasias, including lethal forms. Lethality predictors and the potential of molecular testing are also explored. A structured prenatal approach, combined with timely genetic counselling, may allow for an earlier diagnosis and support informed reproductive decisions. Given the recent advances in reproductive technologies and the potential impact of cartilage-hair hypoplasia on affected individuals, this condition should be actively considered in future studies addressing the prenatal diagnosis of skeletal dysplasias.

#4

Cartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.

Medicine2026 Jan 09

Cartilage-hair hypoplasia (CHH) is an autosomal recessive disorder caused by homozygous or compound heterozygous mutations in the RMRP gene, which is extremely rare in the population, and its most common feature is disproportionately short limb shortness with short and thickened long bones, usually found in newborns, occasionally found in the prenatal stage, and other clinical features include a series of extracutaneous manifestations, such as hypoglycemia, gastrointestinal dysfunction, immunodeficiency, anemia and increased risk of malignant tumors, etc, the specific pathogenesis is unknown. The case of a 1.5-year-old male patient with severe short stature (height 70 cm, <-3 SD), sparse scalp hair, and short limbs, without ligamentous laxity or anemia. Family-based whole-exome sequencing revealed compound heterozygous variants in RMRP: NR_003051.3: n.-21_-9dup and n.5C > T. Classified as pathogenic or likely pathogenic according to ACMG guidelines, these variants confirm the diagnosis of CHH. Due to the elevated tumor predisposition associated with CHH, and because growth hormone therapy is currently contraindicated, no disease-specific interventions have been initiated. We have confirmed that the child has been diagnosed with CHH, but no intervention has been given, and we will do further follow-up and look forward to finding a treatment for the disease. This report describes a rare case of RMRP-associated CHH. This case broadens the clinical understanding of the RMRP mutational spectrum and phenotypic variability, providing new insights into genotype-phenotype correlations in RMRP-associated disorders. We were currently unable to take effective treatment measures for this child, and we hope that in the future, there will be treatments such as gene therapy to bring hope to children and their families.

#5

Refractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.

Journal of pediatric hematology/oncology2026 Mar 01

Cartilage hair hypoplasia-anauxetic dysplasia (CHH-AD) spectrum disorders are rare skeletal dysplasias caused by pathogenic variants in RMRP, associated with immune dysfunction and cancer predisposition. While non-Hodgkin lymphoma is more commonly seen, Hodgkin lymphoma (HL) is rarely reported, and its management in this setting remains unclear. We describe 2 siblings with genetically confirmed CHH-AD who developed relapsed/refractory EBV-positive classic HL. Both presented with short stature, atopy, recurrent infections, and elevated IgE. The brother was diagnosed with stage IV disease, and the sister with stage IIB. Despite receiving frontline chemotherapy, both relapsed within a year. Salvage therapy with gemcitabine/vinorelbine induced metabolic responses, followed by radiotherapy and consolidation with brentuximab vedotin. Autologous transplant was considered but declined by the family due to perceived risks. At 30 months follow-up, both remain in complete remission. Genetic testing confirmed a shared homozygous pathogenic RMRP variant. These cases expand the oncologic spectrum of CHH-AD to include HL, highlight the risk of aggressive disease and early relapse, and demonstrate that durable remission may be achieved without transplantation when consolidation with targeted therapy is feasible. Early recognition of CHH-AD features in HL patients may allow risk-adapted therapy and informed genetic counseling.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC194 artigos no totalmostrando 89

2026

Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.

BMJ case reports
2026

Neonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.

European journal of medical genetics
2026

Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.

Acta medica portuguesa
2026

Cartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.

Medicine
2026

Refractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.

Journal of pediatric hematology/oncology
2025

Precision T cell correction platform for inborn errors of immunity.

Molecular therapy : the journal of the American Society of Gene Therapy
2025

Neonatal erythroderma in a patient with cartilage-hair hypoplasia: Identification of a novel RMRP mutation.

Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG
2025

RMRP variants inhibit the cell cycle checkpoints pathway in cartilage‑hair hypoplasia.

Molecular medicine reports
2025

Anemia in patients with cartilage hair hypoplasia: a narrative review and recommendations.

Laboratory medicine
2024

Identification of a founder effect involving n.197C>T variant in RMRP gene associated to cartilage-hair hypoplasia syndrome in Brazilian patients.

Scientific reports
2024

Cartilage-hair hypoplasia-anauxetic dysplasia spectrum disorders harboring RMRP mutations in two Korean children: A case report.

Medicine
2024

Validation of Risk Factors for Early Mortality in Cartilage-Hair Hypoplasia.

Journal of clinical immunology
2024

Altered oral microbiome, but normal human papilloma virus prevalence in cartilage-hair hypoplasia patients.

Orphanet journal of rare diseases
2024

Prediction of LncRNA-protein Interactions Using Auto-Encoder, SE-ResNet Models and Transfer Learning.

MicroRNA (Shariqah, United Arab Emirates)
2024

Extensive and deep granulomatous ulcers as an atypical manifestation of cartilage-hair hypoplasia syndrome: A diagnostic and therapeutic challenge.

The Australasian journal of dermatology
2024

RMRP-related short stature: A report of six additional Japanese individuals with cartilage hair hypoplasia and literature review.

American journal of medical genetics. Part A
2024

Shorter birth length and decreased T-cell production and function predict severe infections in children with non-severe combined immunodeficiency cartilage-hair hypoplasia.

The journal of allergy and clinical immunology. Global
2023

Long noncoding RNA RMRP ameliorates doxorubicin-induced apoptosis by interacting with PFN1 in a P53-Dependent manner.

Molecular and cellular probes
2023

Japanese siblings with cartilage-hair hypoplasia exhibiting different severity.

Pediatrics international : official journal of the Japan Pediatric Society
2023

Oral findings in patients with cartilage-hair hypoplasia - cross-sectional observational study.

Orphanet journal of rare diseases
2023

Expanding the phenotype of anauxetic dysplasia caused by biallelic NEPRO mutations: A case report.

American journal of medical genetics. Part A
2023

Homozygous RMRP Promoter Duplications Cause Severely Reduced Transcript Abundance and SCID Associated with Cartilage Hair Hypoplasia.

Journal of clinical immunology
2023

Natural history and genetic spectrum of the Turkish metaphyseal dysplasia cohort, including rare types caused by biallelic COL10A1, COL2A1, and LBR variants.

Bone
2022

Lymphomas in cartilage-hair hypoplasia - A case series of 16 patients reveals advanced stage DLBCL as the most common form.

Frontiers in immunology
2022

Reduced Intensity Conditioning Allogeneic Transplant for SCID Associated with Cartilage Hair Hypoplasia.

Journal of clinical immunology
2022

Sirolimus Restores Erythropoiesis and Controls Immune Dysregulation in a Child With Cartilage-Hair Hypoplasia: A Case Report.

Frontiers in immunology
2022

Cartilage-hair hypoplasia with T-cell dysfunction.

Pediatrics international : official journal of the Japan Pediatric Society
2023

Immunologic Heterogeneity in 2 Cartilage-Hair Hypoplasia Patients With a Distinct Clinical Course.

Journal of investigational allergology &amp; clinical immunology
2022

A novel experimental approach for the selective isolation and characterization of human RNase MRP.

RNA biology
2022

A disease-linked lncRNA mutation in RNase MRP inhibits ribosome synthesis.

Nature communications
2021

Uncovering pathways regulating chondrogenic differentiation of CHH fibroblasts.

Non-coding RNA research
2021

Craniofacial and Craniocervical Features in Cartilage-Hair Hypoplasia: A Radiological Study of 17 Patients and 34 Controls.

Frontiers in endocrinology
2021

[Cartilage hair hypoplasia with severe combined immunodeficiency caused by a novel RMRP gene variant].

Zhonghua er ke za zhi = Chinese journal of pediatrics
2021

Long Non-coding RNA RMRP in the Pathogenesis of Human Disorders.

Frontiers in cell and developmental biology
2021

Cartilage-hair Hypoplasia Complicated with Liver Cirrhosis Due to Chronic Intrahepatic Cholestasis.

Internal medicine (Tokyo, Japan)
2022

Partial recapitulation of fetal thymic T-cell constitution postnatally in a patient with cartilage hair hypoplasia-anauxetic dysplasia spectrum disorder: A case report.

Cytometry. Part B, Clinical cytometry
2021

Pulmonary Follow-Up Imaging in Cartilage-Hair Hypoplasia: a Prospective Cohort Study.

Journal of clinical immunology
2021

Crystal structure of human RPP20-RPP25 proteins in complex with the P3 domain of lncRNA RMRP.

Journal of structural biology
2021

Early prenatal presentation of the cartilage-hair hypoplasia / anauxetic dysplasia spectrum of disorders mimicking recurrent thanatophoric dysplasia.

European journal of medical genetics
2021

Homozygous n.64C>T mutation in mitochondrial RNA-processing endoribonuclease gene causes cartilage hair hypoplasia syndrome in two siblings.

European journal of medical genetics
2020

Outcomes of 42 pregnancies in 14 women with cartilage-hair hypoplasia: a retrospective cohort study.

Orphanet journal of rare diseases
2020

How Altered Ribosome Production Can Cause or Contribute to Human Disease: The Spectrum of Ribosomopathies.

Cells
2020

The Safety and Efficacy of Live Viral Vaccines in Patients With Cartilage-Hair Hypoplasia.

Frontiers in immunology
2020

Immunodeficiency in cartilage-hair hypoplasia: Pathogenesis, clinical course and management.

Scandinavian journal of immunology
2020

Rituximab and intense chemotherapy in a patient with defective cell mediated immunity due to cartilage-hair hypoplasia and Burkitt lymphoma.

Pediatric blood &amp; cancer
2020

Identification of Novel and Recurrent RMRP Variants in a Series of Brazilian Patients with Cartilage-Hair Hypoplasia: McKusick Syndrome.

Molecular syndromology
2020

Abnormal Newborn Screening Follow-up for Severe Combined Immunodeficiency in an Amish Cohort with Cartilage-Hair Hypoplasia.

Journal of clinical immunology
2019

[Cartilage-hair hypoplasia. A case report].

Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)
2019

The human long non-coding RNA gene RMRP has pleiotropic effects and regulates cell-cycle progression at G2.

Scientific reports
2020

'Metaphyseal dysplasia without hypotrichosis' can present with late-onset extraskeletal manifestations.

Journal of medical genetics
2019

A 30-Year Prospective Follow-Up Study Reveals Risk Factors for Early Death in Cartilage-Hair Hypoplasia.

Frontiers in immunology
2019

An emerging ribosomopathy affecting the skeleton due to biallelic variations in NEPRO.

American journal of medical genetics. Part A
2019

Rmrp Mutation Disrupts Chondrogenesis and Bone Ossification in Zebrafish Model of Cartilage-Hair Hypoplasia via Enhanced Wnt/β-Catenin Signaling.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
2019

A Systematic Review on Predisposition to Lymphoid (B and T cell) Neoplasias in Patients With Primary Immunodeficiencies and Immune Dysregulatory Disorders (Inborn Errors of Immunity).

Frontiers in immunology
2019

Cutaneous granulomas with primary immunodeficiency in children: a report of 17 new patients and a review of the literature.

Journal of the European Academy of Dermatology and Venereology : JEADV
2019

The antiviral protein viperin regulates chondrogenic differentiation via CXCL10 protein secretion.

The Journal of biological chemistry
2019

Rubella Virus-Associated Cutaneous Granulomatous Disease: a Unique Complication in Immune-Deficient Patients, Not Limited to DNA Repair Disorders.

Journal of clinical immunology
2019

Gynecologic health in cartilage-hair hypoplasia: A survey of 26 adult females.

American journal of medical genetics. Part A
2018

Gynecologic assessment of 19 adult females with cartilage-hair hypoplasia - high rate of HPV positivity.

Orphanet journal of rare diseases
2018

A Wide Spectrum of Autoimmune Manifestations and Other Symptoms Suggesting Immune Dysregulation in Patients With Cartilage-Hair Hypoplasia.

Frontiers in immunology
2018

Anauxetic dysplasia: A rare clinical entity.

The Turkish journal of pediatrics
2018

[Syndromic Hirschsprung&prime;s disease and its mode of inheritance].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2018

Cartilage hair hypoplasia with cutaneous lymphomatoid granulomatosis.

Clinical and experimental dermatology
2018

Diffuse large B-cell lymphoma chemotherapy reveals a combined immunodeficiency syndrome in cartilage hair hypoplasia.

Swiss medical weekly
2018

Diversity of Pubertal Development in Cartilage-Hair Hypoplasia; Two Illustrative Cases.

Journal of pediatric and adolescent gynecology
2018

Quantum Language of MicroRNA: Application for New Cancer Therapeutic Targets.

Methods in molecular biology (Clifton, N.J.)
2017

Expression of RMRP RNA is regulated in chondrocyte hypertrophy and determines chondrogenic differentiation.

Scientific reports
2017

Diverse Autoantibody Reactivity in Cartilage-Hair Hypoplasia.

Journal of clinical immunology
2017

Analysis of clinical and immunologic phenotype in a large cohort of children and adults with cartilage-hair hypoplasia.

The Journal of allergy and clinical immunology
2017

Pregnancy Outcome in Cartilage-Hair Hypoplasia, a Rare Form of Dwarfism.

Case reports in obstetrics and gynecology
2017

Defects in lymphocyte telomere homeostasis contribute to cellular immune phenotype in patients with cartilage-hair hypoplasia.

The Journal of allergy and clinical immunology
2017

Targeted CRISPR disruption reveals a role for RNase MRP RNA in human preribosomal RNA processing.

Genes &amp; development
2017

Cartilage-hair hypoplasia with normal height in childhood-4 patients with a unique genotype.

Clinical genetics
2017

Broadening the phenotypic spectrum of POP1-skeletal dysplasias: identification of POP1 mutations in a mild and severe skeletal dysplasia.

Clinical genetics
2017

Decreased telomere length in children with cartilage-hair hypoplasia.

Journal of medical genetics
2017

Clinical and molecular diagnosis of a cartilage-hair hypoplasia with IGF-1 deficiency.

American journal of medical genetics. Part A
2017

The Finnish founder mutation c.70 A>G in RMRP causes cartilage-hair hypoplasia in a Pakistani family.

Clinical dysmorphology
2016

Hirschsprung's disease associated with alopecia universalis congenita: a case report.

Journal of medical case reports
2017

High prevalence of bronchiectasis in patients with cartilage-hair hypoplasia.

The Journal of allergy and clinical immunology
2016

Further evidence of POP1 mutations as the cause of anauxetic dysplasia.

American journal of medical genetics. Part A
2017

Cartilage-hair hypoplasia associated with isolated hypoganglionosis: A case report.

Congenital anomalies
2016

Hypo- and Hyper-Assembly Diseases of RNA-Protein Complexes.

Trends in molecular medicine
2016

Cartilage Hair Hypoplasia: Two Unrelated Cases with g.70 A > G Mutation in RMRP Gene.

Indian journal of pediatrics
2015

DDX5 and its associated lncRNA Rmrp modulate TH17 cell effector functions.

Nature
2015

Novel Mutation and Structural RNA Analysis of the Noncoding RNase MRP Gene in Cartilage-Hair Hypoplasia.

Molecular syndromology
2015

Cartilage hair hypoplasia: characteristics and orthopaedic manifestations.

Journal of children's orthopaedics
2015

Variable phenotype of severe immunodeficiencies associated with RMRP gene mutations.

Journal of clinical immunology
2015

[Clinical and molecular diagnostics of a cartilage-hair hypoplasia: Two new cases].

Anales de pediatria (Barcelona, Spain : 2003)
2015

Widespread intracranial calcifications in the follow-up of a patient with cartilage-hair hypoplasia--anauxetic dysplasia spectrum disorder: a coincidental finding?

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
Ver todos os 194 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

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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. Familial cartilage-hair hypoplasia: prenatal ultrasound features and clinical outcomes in three siblings with identical RMRP variants.
    BMJ case reports· 2026· PMID 41720498mais citado
  2. Neonatal erythroderma and immunodysplasia: Overlap of cartilage-hair hypoplasia and Omenn syndrome.
    European journal of medical genetics· 2026· PMID 41616907mais citado
  3. Prenatal Diagnosis of Cartilage-Hair Hypoplasia: A Narrative Review.
    Acta medica portuguesa· 2026· PMID 41525162mais citado
  4. Cartilage-hair hypoplasia in a patient with compound heterozygous variants in the RMRP gene: A case report.
    Medicine· 2026· PMID 41517791mais citado
  5. Refractory/Relapsed Hodgkin Lymphoma in Cartilage Hair Hypoplasia-Anauxetic Dysplasia Spectrum: Long-term HSCT-free Remission in 2 Pediatric Siblings.
    Journal of pediatric hematology/oncology· 2026· PMID 41460196mais citado
  6. Expanding the Phenotypic Spectrum of Anauxetic Dysplasia Type 3: Reporting an Iranian Family With Unique Systemic Features and NEPRO Gene Variant.
    Clin Case Rep· 2026· PMID 41982866recente
  7. Successful Treatment of Isolated Bone Marrow Classic Hodgkin Lymphoma in a Patient With Cartilage Hair Hypoplasia.
    Pediatr Blood Cancer· 2026· PMID 41935966recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:175(Orphanet)
  2. OMIM OMIM:250250(OMIM)
  3. MONDO:0009595(MONDO)
  4. GARD:6996(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Q2964438(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

Hipoplasia cartilagem-cabelo
Compêndio · Raras BR

Hipoplasia cartilagem-cabelo

ORPHA:175 · MONDO:0009595
Prevalência
Unknown
Herança
Autosomal recessive
CID-10
Q78.8 · Outras osteocondrodisplasias especificadas
CID-11
Início
Antenatal, Infancy, Neonatal
Prevalência
150.0 (Specific population)
MedGen
UMLS
C0220748
EuropePMC
Wikidata
Papers 10a
DiscussaoAtiva

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