Um grupo de doenças ósseas muito raras, genéticas e progressivas, que causam um tipo específico de baixa estatura, conhecido como nanismo de membros curtos. A baixa estatura acontece porque os antebraços e as canelas são mais curtos que o normal, e os ossos das mãos e dos pés também são encurtados de forma incomum. Ao nascer, as mãos e os pés podem parecer anormalmente curtos e largos. Com o tempo, essa desproporção fica ainda mais evidente, principalmente nos primeiros anos de vida. Outras características podem incluir: dificuldade para esticar totalmente os cotovelos e braços; uma curvatura anormal e progressiva da coluna; uma cabeça maior que o normal; e a parte central do rosto ligeiramente achatada. A Displasia Acromesomélica é uma doença genética. Ela é transmitida quando a pessoa herda uma cópia do gene alterado da mãe e outra do pai, que geralmente não manifestam a doença. Existem diferentes tipos de displasia acromesomélica, que são distinguidos pela sua causa genética. Para saber mais sobre os diferentes tipos, clique nos links abaixo. Displasia Acromesomélica, tipo Maroteaux Displasia Acromesomélica, tipo Hunter-Thompson Displasia Acromesomélica, tipo Grebe
Introdução
O que você precisa saber de cara
A displasia acromesomélica é um grupo raro de condições genéticas que leva a um tipo de nanismo com encurtamento marcante dos antebraços, pernas, mãos e pés. Desde os primeiros anos de vida, as famílias costumam reparar que as mãos e os pés são curtos e largos, além de haver dificuldade para esticar totalmente os braços. A condição é herdada quando a criança recebe os genes alterados de ambos os pais, que geralmente não manifestam nenhuma alteração física. No Brasil, a displasia acromesomélica não possui um protocolo oficial PCDT nem remédios específicos fornecidos pelo SUS, sendo o cuidado focado no suporte ortopédico e na qualidade de vida.
Um grupo de doenças ósseas muito raras, genéticas e progressivas, que causam um tipo específico de baixa estatura, conhecido como nanismo de membros curtos. A baixa estatura acontece porque os antebraços e as canelas são mais curtos que o normal, e os ossos das mãos e dos pés também são encurtados de forma incomum. Ao nascer, as mãos e os pés podem parecer anormalmente curtos e largos. Com o tempo, essa desproporção fica ainda mais evidente, principalmente nos primeiros anos de vida. Outras características podem incluir: dificuldade para esticar totalmente os cotovelos e braços; uma curvatura anormal e progressiva da coluna; uma cabeça maior que o normal; e a parte central do rosto ligeiramente achatada. A Displasia Acromesomélica é uma doença genética. Ela é transmitida quando a pessoa herda uma cópia do gene alterado da mãe e outra do pai, que geralmente não manifestam a doença. Existem diferentes tipos de displasia acromesomélica, que são distinguidos pela sua causa genética. Para saber mais sobre os diferentes tipos, clique nos links abaixo. Displasia Acromesomélica, tipo Maroteaux Displasia Acromesomélica, tipo Hunter-Thompson Displasia Acromesomélica, tipo Grebe
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 64 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 152 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
4 genes identificados com associação a esta condição.
Acromesomelic dysplasia 1
A form of acromesomelic dysplasia, a skeletal disorder characterized by short stature, very short limbs and hand/foot malformations. The severity of limb abnormalities increases from proximal to distal with profoundly affected hands and feet showing brachydactyly and/or rudimentary fingers (knob-like fingers). AMD1 is an autosomal recessive form characterized by axial skeletal involvement with wedging of vertebral bodies. All skeletal elements are present but show abnormal rates of linear growth.
Acromesomelic dysplasia 2A
A form of acromesomelic dysplasia, a skeletal disorder characterized by short stature, very short limbs and hand/foot malformations. The severity of limb abnormalities increases from proximal to distal with profoundly affected hands and feet showing brachydactyly and/or rudimentary fingers (knob-like fingers). AMD2A is an autosomal recessive form characterized by normal axial skeletons and missing or fused skeletal elements within the hands and feet.
Spondylometaphyseal dysplasia, Pagnamenta type
A form of spondylometaphyseal dysplasia, a group of short stature disorders distinguished by abnormalities in the vertebrae and the metaphyses of the tubular bones. SMDP is an autosomal recessive form characterized by short stature and mild platyspondyly with no disproportion between the limbs. Mild metaphyseal changes are present.
Acromesomelic dysplasia 3
A form of acromesomelic dysplasia, a skeletal disorder characterized by short stature, very short limbs and hand/foot malformations. The severity of limb abnormalities increases from proximal to distal with profoundly affected hands and feet showing brachydactyly and/or rudimentary fingers (knob-like fingers). AMD3 is an autosomal recessive form characterized by bilateral aplasia of the fibula, severe brachydactyly, and fusion of carpal and tarsal bones.
Variantes genéticas (ClinVar)
369 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 887 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 — Displasia acromesomélica
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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 12,000-Year-Old Case of NPR2-Related Acromesomelic Dysplasia.
Antenatal unilateral upper limb acromesomelic dysplasia.
Acromesomelic dysplasia (AMD) is an umbrella term given to a heterogeneous group of progressive skeletal disorders characterized by short limbed dwarfism associated with disproportionate shortening of middle and distal segments of the upper as well as lower limbs. Although specific skeletal anomalies are difficult to diagnose antenatally, but because of their antenatal and postnatal implications and a possibility of reoccurrence in following pregnancies, such skeletal anomalies need to be actively addressed. A combination of radiologic, pathologic, genetic and molecular investigation prenatally as well as postnatally is required to classify a specific congenital skeletal dysplasia. Once the genetic make-up of fetal skeletal dysplasia is deciphered, a meaningful genetic counselling could be offered for future pregnancies of affected families. We describe a case of primigravida diagnosed with fetal unilateral upper limb AMD on antenatal ultrasound done at early second trimester. The radius and ulna of left upper limb were abnormally short (less than 5th centile of the mean for that gestational age). The left hand was also hypoplastic. Rest of the sonographic anomaly scan was normal. To the best of our knowledge, AMD limited to unilateral upper limb diagnosed antenatally as an isolated finding is not described in the medical literature so far.
A nonsense mutation in the PRKG2 gene in dalmatian dogs with chondrodysplasia.
Skeletal dysplasias encompass a diverse group of genetic disorders characterized by short stature and dwarfism. In humans, 771 types of skeletal dysplasia have been documented. Similar forms of these disorders have also been observed in dogs. The first cases of documented skeletal dysplasia in Dalmatian dogs were reported in the early 1980s, with additional affected dogs observed in subsequent years. Careful radiological and histopathological examinations at the time revealed severe limb deformities, including shortened radii and ulnae, irregular growth plates and disrupted endochondral ossification. In this study, we applied whole-genome sequencing on samples collected in 1992 and identified a genetic variant in the PRKG2 gene, introducing a premature stop codon (XM_038582312: c.1601T > G, p.L534X). Genetic variants in PRKG2 have previously been implicated in human acromesomelic dysplasia, a disorder affecting limb growth in young children. The PRKG2-encoded protein plays a crucial role in endochondral ossification, and if translated, the identified nonsense variant would result in a truncated protein lacking most of the catalytic domain. Extended screening of the genetic variant revealed its continued segregation in the current Dalmatian population. Furthermore, three recent cases of dwarfism in Dalmatians were found to be homozygous for the identified PRKG2 nonsense variant. These findings provide compelling evidence for the role of PRKG2 in Dalmatian dwarfism, resolving a decades-old genetic mystery in the breed.
A novel variant in NPR2: C.2291T > C (p.Leu764Pro) identified in a patient with acromesomelic dysplasia Maroteaux type.
Acromesomelic dysplasia Maroteaux type (AMDM) is a rare autosomal recessive skeletal dysplasia with an estimated prevalence of 1:1,000,000. It is characterized by extreme shortening of the forelimbs and disproportionate short stature. Here we present the clinical and genetic features of an 8-year-8-month-old boy exhibiting idiopathic short stature and abnormal changes of the appendicular skeleton and axial skeleton, consistent with the established phenotypic spectrum of AMDM. Using diagnostic exome sequencing, we identified two variants in NPR2: a known pathogenic nonsense variant, C.2965 C > T (p.Arg989*), and a missense variant of unknown significance, C.2291T > C (p.Leu764Pro), which has never been reported before. Sanger sequencing confirmed that the variants were inherited from his phenotypically normal parents. The proband is compound heterozygous, while both parents are heterozygous carriers, indicating an autosomal recessive pattern of inheritance. This study enriches the pathogenic gene mutation spectrum of NPR2 in patients with AMDM and further emphasizes the application of molecular genetic detection in the diagnosis of rare skeletal abnormalities.
Case Report: Dual pathogenic mechanism of a PRKG2 missense variant underlies an attenuated phenotype of acromesomelic dysplasia.
Acromesomelic dysplasia comprises a group of rare skeletal disorders characterized by dwarfism with anomalies predominantly affecting the middle and distal segments of the limbs. Based on genetic variants, five types are recognized, with significant phenotypic variability even within a single type. Here, we describe a girl presenting with borderline short stature and mild disproportion due to acro- and mesomelic shortening of the limbs. Radiographic examination revealed shortening of the radius and ulna, mild brachydactyly, absence of iliac flaring and metaphyseal alterations of the long bones, and biconcave appearance of the femoral necks and II-IV metacarpals, and an elongated styloid process of the ulna. Using WGS, we identified two novel variants in the PRKG2 gene: a frameshift variant (NM_006259.3:c.1074del (p.Ala359LeufsTer24)) and a missense variant (NM_006259.3:c.1630G>T (p.Asp544Tyr)). Functional analysis unveiled a unique dual pathogenic mechanism: the missense variant creates a cryptic splice site, resulting in two aberrant protein products - an in-frame deletion and a missense substitution. We hypothesize that these alterations cause a partial, rather than a complete, loss of protein function, which may account for the patient's attenuated clinical phenotype.
Publicações recentes
A 12,000-Year-Old Case of NPR2-Related Acromesomelic Dysplasia.
Case Report: Dual pathogenic mechanism of a PRKG2 missense variant underlies an attenuated phenotype of acromesomelic dysplasia.
A nonsense mutation in the PRKG2 gene in dalmatian dogs with chondrodysplasia.
Management of a geminated tooth and supernumeraries in a patient with Acromesomelic Dysplasia, Maroteaux type.
A novel variant in NPR2: C.2291T > C (p.Leu764Pro) identified in a patient with acromesomelic dysplasia Maroteaux type.
📚 EuropePMC64 artigos no totalmostrando 57
A 12,000-Year-Old Case of NPR2-Related Acromesomelic Dysplasia.
The New England journal of medicineCase Report: Dual pathogenic mechanism of a PRKG2 missense variant underlies an attenuated phenotype of acromesomelic dysplasia.
Frontiers in geneticsA nonsense mutation in the PRKG2 gene in dalmatian dogs with chondrodysplasia.
PloS oneManagement of a geminated tooth and supernumeraries in a patient with Acromesomelic Dysplasia, Maroteaux type.
Journal of clinical orthodontics : JCOA novel variant in NPR2: C.2291T > C (p.Leu764Pro) identified in a patient with acromesomelic dysplasia Maroteaux type.
Italian journal of pediatricsAcromesomelic Dysplasia With Homozygosity for a Likely Pathogenic BMPR1B Variant: Postaxial Polydactyly as a Novel Clinical Finding.
Molecular genetics & genomic medicineAntenatal unilateral upper limb acromesomelic dysplasia.
Journal of ultrasoundCongenital hallux valgus occurs in Fibrodysplasia Ossificans Progressiva and BMPR1B-associated dysplasia: an important distinction.
BMC medical genomicsAnaesthetic challenges in a patient with acromesomelic dysplasia posted for vitreoretinal surgery - A case study.
Indian journal of anaesthesiaUnveiling the pathogenic mechanisms of NPR2 missense variants: insights into the genotype-associated severity in acromesomelic dysplasia and short stature.
Frontiers in cell and developmental biologyTwo new patients with acromesomelic dysplasia, PRKG2 type-identification and characterization of the first missense variant.
European journal of human genetics : EJHGA novel variant in BMPR1B causes acromesomelic dysplasia Grebe type in a consanguineous Moroccan family: Expanding the phenotypic and mutational spectrum of acromesomelic dysplasias.
BoneEvaluation of polysomnography findings in children with genetic skeletal disorders.
Journal of sleep researchFibular Agenesis and Ball-Like Toes Mimicking Preaxial Polydactyly: Prenatal Presentation of Du Pan Syndrome.
Molecular syndromologyNatural history of clinical features in two brothers with acromesomelic dysplasia related to PRKG2.
Clinical geneticsIdentification of Diagnostic Variants in FGFR2 and NPR2 Genes in a Chinese Family Affected by Crouzon Syndrome and Acromesomelic Dysplasia, Type Maroteaux.
DNA and cell biologyWDR35 variants in a cranioectodermal dysplasia patient with early onset end-stage renal disease and retinal dystrophy.
American journal of medical genetics. Part ABiallelic KIF24 Variants Are Responsible for a Spectrum of Skeletal Disorders Ranging From Lethal Skeletal Ciliopathy to Severe Acromesomelic Dysplasia.
Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral ResearchNovel NPR2 Gene Mutations Affect Chondrocytes Function via ER Stress in Short Stature.
CellsNovel Loss-of-Function Mutations in NPR2 Cause Acromesomelic Dysplasia, Maroteaux Type.
Frontiers in geneticsNPR2 gene variants in familial short stature: a single-center study.
Journal of pediatric endocrinology & metabolism : JPEMClinical relevance of targeted exome sequencing in patients with rare syndromic short stature.
Orphanet journal of rare diseasesA mild case of acromesomelic dysplasia, type Maroteaux with novel natriuretic peptide receptor B (NPR2) variants.
Radiology case reportsAcromesomelic dysplasia-Maroteaux type, nine patients with two novel NPR2 variants.
Journal of pediatric endocrinology & metabolism : JPEMRare case of dual diagnosis in consanguineous family: a case report.
Clinical dysmorphologyPhosphatase inhibition by LB-100 enhances BMN-111 stimulation of bone growth.
JCI insightGeneration of induced pluripotent stem cell line (IGIBi007-A) from a patient with a novel acromesomelic dysplasia, PRKG2 type (AMDP).
Stem cell researchA case report of Arnold Chiari type 1 malformation in acromesomelic dwarf infant.
The Pan African medical journalFurther defining the clinical and molecular spectrum of acromesomelic dysplasia type maroteaux: a Turkish tertiary center experience.
Journal of human geneticsAcromesomelic Dysplasia, Type Maroteaux: Impact of Long-Term (8 Years) High-Dose Growth Hormone Treatment on Growth Velocity and Final Height in 2 Siblings.
Hormone research in paediatricsBiallelic cGMP-dependent type II protein kinase gene (PRKG2) variants cause a novel acromesomelic dysplasia.
Journal of medical geneticsShort Stature is Progressive in Patients with Heterozygous NPR2 Mutations.
The Journal of clinical endocrinology and metabolismA novel NPR2 mutation (p.Arg388Gln) in a patient with acromesomelic dysplasia, type Maroteaux.
Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric EndocrinologyA novel nonsense mutation in NPR2 gene causing Acromesomelic dysplasia, type Maroteaux in a consanguineous family in Southern Punjab (Pakistan).
Genes & genomicsSkeletal ciliopathies: a pattern recognition approach.
Japanese journal of radiologyAn Iranian Patient with Maroteaux Type Acromesomelic Dysplasia, Showing no Involvement of Distal Lower Limbs.
Journal of clinical research in pediatric endocrinologyAcromesomelic dysplasia Maroteaux-type in patients from Vietnam.
American journal of medical genetics. Part AHeterozygous NPR2 Mutation in Two Family Members with Short Stature and Skeletal Dysplasia.
Case reports in endocrinologyNovel variants in natriuretic peptide receptor 2 in unrelated patients with acromesomelic dysplasia type Maroteaux.
European journal of medical geneticsBone morphogenetic protein receptor signal transduction in human disease.
The Journal of pathologyMolecular and in silico analyses validates pathogenicity of homozygous mutations in the NPR2 gene underlying variable phenotypes of Acromesomelic dysplasia, type Maroteaux.
The international journal of biochemistry & cell biologyLinked homozygous BMPR1B and PDHA2 variants in a consanguineous family with complex digit malformation and male infertility.
European journal of human genetics : EJHGIdentification of one novel homozygous mutation in the NPR2 gene in a patient from Taiwan with acromesomelic dysplasia Maroteaux type.
Pediatrics and neonatologyA novel homozygous variant in BMPR1B underlies acromesomelic dysplasia Hunter-Thompson type.
Annals of human geneticsNew pathogenic variant in the NPR2 gene: Etiology of low size, macrocephaly and bone dysplasia in a male with acromesomelic dysplasia Maroteaux-type.
Medicina clinicaNovel mutations in the transmembrane natriuretic peptide receptor NPR-B gene in four Indian families with acromesomelic dysplasia, type Maroteaux.
Journal of geneticsNovel homozygous sequence variants in the GDF5 gene underlie acromesomelic dysplasia type-grebe in consanguineous families.
Congenital anomaliesNew insights into the structure, assembly and biological roles of 10-12 nm connective tissue microfibrils from fibrillin-1 studies.
The Biochemical journalGenetics of human isolated acromesomelic dysplasia.
European journal of medical geneticsRecurrent mutation in CDMP1 in a family with Grebe chondrodysplasia: broadening the phenotypic manifestation of syndrome in Pakistani population.
Pakistan journal of medical sciencesAcromesomelic dysplasia, type maroteaux caused by novel loss-of-function mutations of the NPR2 gene: Three case reports.
American journal of medical genetics. Part AA NOVEL MUTATION IN NPR2 GENE IN A PATIENT WITH ACROMESOMELIC DYSPLASIA, MAROTEAUX TYPE.
Genetic counseling (Geneva, Switzerland)Characterization of an acromesomelic dysplasia, Grebe type case: novel mutation affecting the recognition motif at the processing site of GDF5.
Journal of bone and mineral metabolismA hypomorphic BMPR1B mutation causes du Pan acromesomelic dysplasia.
Orphanet journal of rare diseasesHomozygous sequence variants in the NPR2 gene underlying Acromesomelic dysplasia Maroteaux type (AMDM) in consanguineous families.
Annals of human geneticsAccommodating difference in the prehistoric past: Revisiting the case of Romito 2 from a bioarchaeology of care perspective.
International journal of paleopathologyHeterozygous mutations in natriuretic peptide receptor-B (NPR2) gene as a cause of short stature.
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Doença com base genética
Um médico geneticista pode ajudar no diagnóstico de Displasia acromesomélica 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
Ela é uma doença genética transmitida quando a pessoa herda uma alteração nos genes de ambos os pais, que habitualmente não apresentam a condição. As alterações genéticas identificadas envolvem os genes NPR2, GDF5, PRKG2 e BMPR1B.
Referências
Fontes citadas no texto, publicações do grafo e bases de dados usadas neste verbete
6 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.). Displasia acromesomélica. Em Raras — Enciclopédia de Doenças Raras do Brasil. https://raras.org/doenca/displasia-acromesomelica
Formato APA. Conteúdo sob CC BY 4.0 — reuso livre com atribuição.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
Displasia acromesomélica
📋 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
- CID-11 (futuro)
- fonte: WHO ICD-11
- NIH/GARD
- fonte: GARD (NIH)
- Indexação biomédica
- fonte: MeSH (NLM)
- Dado público estruturado
- fonte: Wikidata