Development of domagrozumab was discontinued despite the fact that treated participants showed a trend of increasing muscle mass volume, assessed by MRI, and improvement on the North Star Ambulatory Assessment (NSAA) score ( Wagner et al., 2020 ).
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Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy.
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The sentences
Ultimately, the difference between drisapersen-treated (with 6 mg/kg/week) and placebo-treated patients did not reach statistical significance on the 6MWT at 48 weeks or on key secondary endpoints.