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Duchenne muscular dystrophy

Does randomization matter in rare disease clinical trials

Stephanie Wied, Denis Kirilov Razsolkov, Nicole Heussen, Ralf-Dieter Hilgers

published 02 Sep 2026

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Barriers and levers to using an app for motor function assessment by therapists in hospitals: A qualitative study of the MFM-Play app

Dominique Vincent-Genod, Manon Verroul, Pascal Rippert, Shotaro Tachibana, Carole Vuillerot, MFM-Play Study Group

published 31 Jul 2026

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Accuracy of the molecular diagnosis of duchenne and becker muscular dystrophy: A systematic review with meta-analysis

Fabian A. Chavez-Ecos, Carlos Quispe-Vicuña, Marco Malaga, Niels Pacheco-Barrios, Andrely Huerta-Rosario, Victor Velasquez-Rimachi, Peggy Martinez Esteban, Carlos Alva-Diaz

published 21 Jul 2026

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A natural history study of Chinese individuals with Duchenne muscular dystrophy: Results from 2 years of follow-up and beyond

Xihua Li, Xinying Yang, Xingzhi Chang, Zhiqiang Wang, Siqi Hong, Wenhua Zhu, Qi Shen, Yangmei Zhou, Shuiqing Zhu, Ying Xu Gao, Caiping Jia

published 02 Apr 2026

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A shape-based functional index for objective assessment of pediatric motor function

Shashwat Kumar, Arafat Rahman, Robert Gutierrez, Sarah Livermon, Allison N. McCrady, Silvia Blemker, Rebecca Scharf, Anuj Srivastava, Laura E. Barnes

published 10 Oct 2025

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Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy

Francesco Muntoni, James Signorovitch, Nathalie Goemans, Adnan Y. Manzur, Nicolae Done, Gautam Sajeev, Jiayang Li, Hanane Akbarnejad, Aarushi Sharma, Susan J. Ward, Erik H. Niks, Laurant Servais, Eugenio Mercuri, Michela Guglieri, Volker Straub, Imelda de Groot, Deborah Ridout, PRO-DMD-01 study investigators , Association Française contre les Myopathies , The NorthStar Clinical Network , Craig McDonald

published 27 Jun 2025

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The prospective acceptability of preventative IV bisphosphonate therapy prior to fracture: Perspectives of young people with Duchenne muscular dystrophy, parents and health professionals

Danielle C. Mountain, Nicola Crabtree, Claire L. Wood, Sze Choong Wong, Lucy Bray

published 02 Jun 2025

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Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy

Ines Vandekerckhove, Marleen Van den Hauwe, Tijl Dewit, Geert Molenberghs, Nathalie Goemans, Liesbeth De Waele, Anja Van Campenhout, Friedl De Groote, Kaat Desloovere

published 18 Mar 2025

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Identification of suitable qPCR reference genes for the normalization of gene expression in the BL10-mdx and D2-mdx mouse models of Duchenne muscular dystrophy

Kayleigh Putker, Anne-Fleur Schneider, Davy Van De Vijver, John Hildyard, Annemieke Aartsma-Rus, Maaike van Putten

published 25 Feb 2025

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Respiratory pathology in the mdx/utrn ^-/- mouse: A murine model for Duchenne Muscular Dystrophy (DMD)

Marán Y. Hernández Rodríguez, Debolina D. Biswas, Aoife D. Slyne, Jane Lee, Evelyn Scarrow, Sarra M. Abdelbarr, Heather Daniels, Ken D. O’Halloran, Leonardo F. Ferreira, Charles A. Gersbach, Mai K. ElMallah

published 07 Feb 2025

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Navigating adulthood: Exploring the transition needs of adolescents and young adults affected by Duchenne or Becker muscular dystrophy

Sharon Barak, Shirley Ackerman Laufer, Michal Gudinsky Elyashiv, Sharon Rubinstein-Shatz, Rama Davidson, Tali Kaplan

published 14 Jan 2025

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Height development and multiple bone health indicators in children aged 2–12 years with Duchenne muscular dystrophy (DMD)

Bingying Wang, Linyuhan Zhou, Shuangru Li, Huayan Xu, Yingkun Guo, Qin Hu, Min Huang, Dan Zhou, Xiaotang Cai, Qiu Wang, Xiaomei Sun

published 10 Jan 2025

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Determination of qPCR reference genes suitable for normalizing gene expression in a novel model of Duchenne muscular dystrophy, the D2-mdx mouse

Brigida Boccanegra, Roberta Lenti, Paola Mantuano, Elena Conte, Lisamaura Tulimiero, Richard J. Piercy, Ornella Cappellari, John C. W. Hildyard, Annamaria De Luca

published 13 Nov 2024

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