Uma síndrome que se caracteriza por atraso no desenvolvimento psicomotor, cabeça com formato mais curto ou achatado na parte de trás e um rosto plano, nariz pequeno, boca pequena, fenda no céu da boca, catarata, perda auditiva, saco escrotal pouco desenvolvido e alterações nos dedos (das mãos e dos pés).
Introdução
O que você precisa saber de cara
Uma síndrome que se caracteriza por atraso no desenvolvimento psicomotor, cabeça com formato mais curto ou achatado na parte de trás e um rosto plano, nariz pequeno, boca pequena, fenda no céu da boca, catarata, perda auditiva, saco escrotal pouco desenvolvido e alterações nos dedos (das mãos e dos pés).
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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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
+ 26 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 81 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
1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.
Acts as a transcriptional activator or repressor. Involved in embryonic lens fiber cell development. Recruits the transcriptional coactivators CREBBP and/or EP300 to crystallin promoters leading to up-regulation of crystallin gene during lens fiber cell differentiation. Activates the expression of IL4 in T helper 2 (Th2) cells. Increases T-cell susceptibility to apoptosis by interacting with MYB and decreasing BCL2 expression. Together with PAX6, transactivates strongly the glucagon gene promote
Nucleus
Variantes genéticas (ClinVar)
136 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 100 variantes classificadas pelo 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 — Síndrome Aymé-Gripp
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
A Novel MAF Variant Causing Aymé-Gripp Syndrome With Transient Anemia.
Impedance-based detection of cochlear implant array migration: case report in a child with Aymé-Gripp syndrome.
Detection of complications during rehabilitation and postoperative follow-up after cochlear implantation is essential, especially in children and cognitively impaired patients. Electrode array migration can affect outcomes and must be detected early. Traditional radiographic methods, although effective, are costly and expose patients to radiation. This case report discusses the use of a previously published impedance-based model for cochlear implant array localization in a child with Aymé-Gripp syndrome. Impedance telemetry data and X-ray images were collected at the time of initial surgery and before and after the required revision surgery. The impedance-based model was used to estimate the insertion depth of the most basal cochlear implant electrode within the cochlea. The resulting estimates were compared with the electrode positions from radiographs to assess the accuracy and applicability of the model. 20 months after implantation, the patient suddenly stopped tolerating the CI audio processor. Retrospectively, the impedance-based model revealed substantial electrode migration, which was confirmed by postoperative radiography. The proposed model, which uses routine impedance telemetry data without radiation exposure, offers a cost-effective alternative to radiography. Early detection and intervention, particularly in complex cases, improves outcomes and reduces costs, highlighting the importance of objective monitoring.
Comparison of Genetic, Auditory Features, and Systemic Clinical Phenotype in 14 Families with Syndromic Hearing Loss.
Syndromic hearing loss (SHL) is characterized by distinctive clinical phenotypes as well as genetic and phenotypic heterogeneity. More than 400 species of SHL have been described, the majority of which are autosomal dominant. 11 forms of SHL were obtained from 14 unrelated families with probands ranging in age from 5 to 78 months. The results of whole exome sequencing(WES), audiological characteristics, middle and inner ear radiological findings, and additional clinical phenotype characteristics were retrospectively analyzed. Fourteen people with SHL were found. Two of them had Waardenburg syndrome, two had Branchio-Oto-Renal syndrome, two had CHARGE syndrome, and one had Treacher Collins syndrome, Kleefstra syndrome, Muenke syndrome, Osteopathia Striata with Cranial Sclerosis, Ayme-Gripp syndrome, Tatton-Brown-Rahman syndrome, Stickler syndrome, or Stapes Ankylosis with Broad Thumbs and Toes. In this investigation, ten variants were first reported. The combination of a neonatal hearing screening and WES can diagnose syndrome-type hearing loss in infancy and childhood, according to our findings, expansion of the gene variant spectrum and phenotype for various age groups of SHL is essential and can provide valuable guidelines for clinical intervention decisions. It is imperative for medical practitioners to conduct diligent and prolonged patient monitoring due to the inherent variability in both the auditory impairment and the comprehensive clinical manifestation of SHL.
Massive pericardial effusion in an infant with Aymé-Gripp syndrome: A case report and review of the literature.
Aymé-Gripp syndrome (AYGRPS) is a multisystemic disorder caused by a subset of pathogenic variants in the MAF gene. Major clinical features include bilateral early cataracts, sensorineural hearing loss (SNHL), and a characteristic facial appearance along with variable neurodevelopmental delay. Pericarditis resulting in pericardial effusion of varying degree has been observed in a subset of affected individuals and could represent a severe feature in neonatal or infantile age. Here, we describe a syndromic infant with massive pericardial effusion and craniofacial features that oriented toward the suspicion of AYGRPS, which was subsequently confirmed by the molecular analysis of MAF. Pericardial effusion was first observed prenatally and documented to be recurrent, progressive, and severe in the first months of life, thus requiring pericardiocentesis and surgical procedures. In this report, we provide further delineation of the minor clinical characteristics, particularly focusing on cardiac features of AYGRPS. A dedicated cardiac surveillance of these findings may help reduce the morbidity and mortality of this rare condition.
Case report: Whole exome sequencing identified a novel mutation (p.Y301H) of MAF in a Chinese family with congenital cataracts.
Congenital cataracts stand as the primary cause of childhood blindness globally, characterized by clouding of the eye's lens at birth or shortly thereafter. Previous investigations have unveiled that a variant in the V-MAF avian musculoaponeurotic-fibrosarcoma oncogene homolog (MAF) gene can result in Ayme-Gripp syndrome and solitary cataract. Notably, MAF mutations have been infrequently reported in recent years. In this investigation, we recruited a Chinese family with non-syndromic cataracts. Whole exome sequencing and Sanger sequencing were applied to scrutinize the genetic anomaly within the family. Through whole exome sequencing and subsequent data filtration, a new mutation (NM_005360, c.901T>C/p.Y301H) in the MAF gene was detected. Sanger sequencing validated the presence of this mutation in another affected individual. The p.Y301H mutation, situated in an evolutionarily preserved locus, was not detected in our 200 local control cohorts and various public databases. Additionally, multiple bioinformatic programs predicted that the mutation was deleterious and disrupted the bindings between MAF and its targets. Hence, we have documented a new MAF mutation within a Chinese family exhibiting isolated congenital cataracts. Our study has the potential to broaden the spectrum of MAF mutations, offering insights into the mechanisms underlying cataract formation and facilitating genetic counseling and early diagnosis for congenital cataract patients.
Publicações recentes
A Novel MAF Variant Causing Aymé-Gripp Syndrome With Transient Anemia.
Impedance-based detection of cochlear implant array migration: case report in a child with Aymé-Gripp syndrome.
Comparison of Genetic, Auditory Features, and Systemic Clinical Phenotype in 14 Families with Syndromic Hearing Loss.
Massive pericardial effusion in an infant with Aymé-Gripp syndrome: A case report and review of the literature.
Case report: Whole exome sequencing identified a novel mutation (p.Y301H) of MAF in a Chinese family with congenital cataracts.
📚 EuropePMC12 artigos no totalmostrando 19
A Novel MAF Variant Causing Aymé-Gripp Syndrome With Transient Anemia.
Congenital anomaliesImpedance-based detection of cochlear implant array migration: case report in a child with Aymé-Gripp syndrome.
European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck SurgeryComparison of Genetic, Auditory Features, and Systemic Clinical Phenotype in 14 Families with Syndromic Hearing Loss.
The application of clinical geneticsMassive pericardial effusion in an infant with Aymé-Gripp syndrome: A case report and review of the literature.
American journal of medical genetics. Part ACase report: Whole exome sequencing identified a novel mutation (p.Y301H) of MAF in a Chinese family with congenital cataracts.
Frontiers in medicineEctoderm-derived findings in Aymè-Gripp syndrome.
The Journal of dermatologyAn atypical Aymé-Gripp phenotype detected by exome sequencing.
American journal of medical genetics. Part AGeneration and mutational analysis of a transgenic murine model of the human MAF mutation.
American journal of medical genetics. Part ABroadening the genotypic and phenotypic spectrum of MAF in three Chinese Han congenital cataracts families.
American journal of medical genetics. Part ACharacteristics and outcomes of glomerulonephritis with membranoproliferative pattern in children.
Translational pediatricsIsolated cytokine-enriched pericardial effusion: A likely key feature for Aymé-Gripp syndrome.
American journal of medical genetics. Part AUltra-rare renal diseases diagnosed with whole-exome sequencing: Utility in diagnosis and management.
BMC medical genomicsAyme gripp syndrome in an Indian patient.
American journal of medical genetics. Part ASkeletal abnormalities are common features in Aymé-Gripp syndrome.
Clinical geneticsMaternally inherited MAF variant associated with variable expression of Aymé-Gripp syndrome.
American journal of medical genetics. Part AFurther delineation of Aymé-Gripp syndrome and use of automated facial analysis tool.
American journal of medical genetics. Part AMutation update of transcription factor genes FOXE3, HSF4, MAF, and PITX3 causing cataracts and other developmental ocular defects.
Human mutationNovel missense mutation in the bZIP transcription factor, MAF, associated with congenital cataract, developmental delay, seizures and hearing loss (Aymé-Gripp syndrome).
BMC medical geneticsMutations Impairing GSK3-Mediated MAF Phosphorylation Cause Cataract, Deafness, Intellectual Disability, Seizures, and a Down Syndrome-like Facies.
American journal of human geneticsAssociações
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 Síndrome Aymé-Gripp
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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.
- A Novel MAF Variant Causing Aymé-Gripp Syndrome With Transient Anemia.
- Impedance-based detection of cochlear implant array migration: case report in a child with Aymé-Gripp syndrome.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery· 2025· PMID 40301235mais citado
- Comparison of Genetic, Auditory Features, and Systemic Clinical Phenotype in 14 Families with Syndromic Hearing Loss.
- Massive pericardial effusion in an infant with Aymé-Gripp syndrome: A case report and review of the literature.
- Case report: Whole exome sequencing identified a novel mutation (p.Y301H) of MAF in a Chinese family with congenital cataracts.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:1272(Orphanet)
- OMIM OMIM:601353(OMIM)
- MONDO:0011049(MONDO)
- GARD:958(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Q55783094(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