PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular...( - Findings Suggest Clinically Mea...),Health

- Findings Suggest Clinically Meaningful Primary Endpoint for Clinical Trials; Data Presented at the 13th International World Muscle Society Congress -

SOUTH PLAINFIELD, N.J., Sept. 30 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced promising findings in patients with Duchenne/Becker Muscular Dystrophy (DMD/BMD) at the 13th International World Muscle Society (WMS) Congress. Results from an observational study assessing the utility of the 6-minute walk test (6MWT) as a primary outcome measure in trials of treatment for DMD/BMD showed that the 6MWT clearly differentiates boys with DMD from healthy boys, especially when adjusted for age. The findings also demonstrate that the 6MWT provides a reliable assessment of ambulatory function in boys as young as five years of age, offering a practical and clinically meaningful outcome measure for use in DMD/BMD registration-directed clinical trials. Results from the study were presented today by Craig McDonald, M.D., principal investigator and director of the Rehabilitation Research and Training Center in Neuromuscular Diseases, University of California Davis.

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There has been a strong interest in establishing clinically meaningful outcomes measures to advance the development of new treatments for DMD/BMD. The 6MWT is an accepted and standardized measure of ambulatory capacity that was developed to provide an integrated assessment of cardiac, respiratory, circulatory and muscular capacity. Until this study, it had been unclear whether boys with DMD would have the stamina and mental focus to successfully finish the test without injury. This study demonstrated that ambulatory boys with DMD as young as five years old can complete the 6MWT safely and reliably with minimal interruption from falls. The study found that the muscular deficits that are chara
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