MedElevator Войти в исследования
← Поиск публикаций

DOI 10.1002/ajmga.70046

Медицинская библиотека · American journal of medical genetics. Part A · 2026

Expanding the Genotype-Phenotype Correlation of Marden-Walker Syndrome due to PIEZO2 Gene Variants: A Case Report From Brazil.

Battiston Guilherme Sotto, Araujo Carolina de Souza, Romera Fernanda Araujo, Ferreira André Luis, Martins Érica Trovisco, de Aguiar Carolina Galhós, Santos José Eduardo Mourão, Ragazzini Rodrigo, Costa-Nobre Daniela Testoni, Prestes Ana Claudia Yoshikumi, de Oliveira Allan Chiaratti, Perrone Eduardo, Melo Débora Gusmão

Клинический случайСтатьяПоддержка исследования: вне правительства СШАПеревод в очереди

Аннотация

Marden-Walker syndrome (MWS; OMIM 248700) is an extremely rare congenital disorder characterized by multiple joint contractures, craniofacial dysmorphism, neurological abnormalities, and multisystem involvement. Although historically diagnosed on clinical grounds, only a few cases have been molecularly confirmed. Here, we describe a Brazilian female infant with classic manifestations of MWS, carrying a heterozygous pathogenic variant in the PIEZO2 gene not previously reported in MWS. To our knowledge, this is the first molecularly confirmed MWS case from Brazil, thus expanding both the genotype-phenotype spectrum and geographic distribution of PIEZO2-related disorders. Comparative analysis of previously reported molecularly confirmed cases reveals shared core features and highlights the prominent neurological involvement observed in our patient. A review of individuals with the same PIEZO2 variant demonstrates marked phenotypic variability-from Gordon syndrome to distal arthrogryposis type 5-underscoring allelic heterogeneity and variable expressivity. This case refines the phenotypic spectrum of PIEZO2-related disorders and illustrates how allelic heterogeneity contributes to wide clinical variability, while also underscoring the importance of including underrepresented populations in variant interpretation.

Для цитирования: Battiston Guilherme Sotto, Araujo Carolina de Souza, Romera Fernanda Araujo, Ferreira André Luis, Martins Érica Trovisco, de Aguiar Carolina Galhós, Santos José Eduardo Mourão, Ragazzini Rodrigo, Costa-Nobre Daniela Testoni, Prestes Ana Claudia Yoshikumi, de Oliveira Allan Chiaratti, Perrone Eduardo, Melo Débora Gusmão. Expanding the Genotype-Phenotype Correlation of Marden-Walker Syndrome due to PIEZO2 Gene Variants: A Case Report From Brazil.. American journal of medical genetics. Part A. 2026. DOI: 10.1002/ajmga.70046.

Предметные рубрики MeSH

Подготовка и перевод публикации
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…

История · было → стало

Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.

Тип публикации указан по данным источника. Уровень доказательности автоматически не присваивается.