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Muscular dystrophies

Does randomization matter in rare disease clinical trials

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

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, [ ... ], MFM-Play Study Group

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

Fabian A. Chavez-Ecos, Carlos Quispe-Vicuña, [ ... ], Carlos Alva-Diaz

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

Xihua Li, Xinying Yang, [ ... ], Caiping Jia

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

Shashwat Kumar, Arafat Rahman, [ ... ], Laura E. Barnes

Effectiveness of the capability approach in rehabilitation for persons with neuromuscular diseases: A controlled before-after study

Eirlys J. Pijpers, Bart Bloemen, [ ... ], Edith H. C. Cup

Clinical outcomes in patients with muscle disorders and acute ischemic stroke

Adeel S. Zubair, Lauren Littig, Daniel B. DiCapua, Adam de Havenon

3D finite element models reveal regional fatty infiltration modulates tibialis anterior force generating capacity in FSHD

Allison McCrady, Seth Friedman, [ ... ], Silvia Blemker

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

Francesco Muntoni, James Signorovitch, [ ... ], Craig McDonald

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, [ ... ], Lucy Bray

Unraveling genetic etiologies in complex pediatric neurological diseases: A genetic investigation using whole exome sequencing

Zainab Gaouzi, Aziza Belkhayat, [ ... ], Yamna Kriouile

Deep learning-based acceleration of muscle water T2 mapping in patients with neuromuscular diseases by more than 50% - translating quantitative MRI from research to clinical routine

Joachim Schmitt, Dominik Weidlich, [ ... ], Sarah Schlaeger

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

Ines Vandekerckhove, Marleen Van den Hauwe, [ ... ], Kaat Desloovere

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