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Duchenne muscular dystrophy

Comprehensive genetic analysis of 961 unrelated Duchenne Muscular Dystrophy patients: Focus on diagnosis, prevention and therapeutic possibilities

Shalini H. Kumar, Kalpana Athimoolam, [ ... ], Lakshmi Bremadesam Raman

Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration

Nathalie Goemans, Brenda Wong, [ ... ], the Collaborative Trajectory Analysis Project (cTAP)

Urinary incontinence in men with Duchenne and Becker muscular dystrophy

Christopher I. Morse, Katie Higham, Emma L. Bostock, Matthew F. Jacques

A new linear regression-like residual for survival analysis, with application to genome wide association studies of time-to-event data

Veronica J. Vieland, Sang-Cheol Seok, William C. L. Stewart

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmdmdx rat model

Dorian Caudal, Virginie François, [ ... ], Corinne Huchet

Evaluation of the cognitive-motor performance of adults with Duchenne Muscular Dystrophy in a hand-related task

Kostas Nizamis, Wouter Schutte, [ ... ], Bart F. J. M. Koopman

Diagnostic differences in respiratory breathing patterns and work of breathing indices in children with Duchenne muscular dystrophy

Lauren Ryan, Tariq Rahman, [ ... ], Thomas H. Shaffer

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

Francesco Muntoni, Joana Domingos, [ ... ], Susan J. Ward

Investigating the clinical use of structured light plethysmography to assess lung function in children with neuromuscular disorders

Deborah Fleck, Chistopher Curry, [ ... ], Ciara M. Hughes

Discovery of potential urine-accessible metabolite biomarkers associated with muscle disease and corticosteroid response in the mdx mouse model for Duchenne

Mathula Thangarajh, Aiping Zhang, [ ... ], Simina M. Boca

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Claudia Brogna, Giorgia Coratti, [ ... ], on behalf on the International DMD group

A standardised framework to identify optimal animal models for efficacy assessment in drug development

Guilherme S. Ferreira, Désirée H. Veening-Griffioen, [ ... ], Peter J. K. van Meer

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

Holly Landrum Peay, Ryan Fischer, [ ... ], Carol Mansfield

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