Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids.

Neuromuscular Disorders(2017)

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摘要
•Comparison of drisapersen-treated with untreated matched natural history subjects.•Matching was performed by baseline age (±6 mo) and 6MWD (±30 m) as primary analysis.•Matching was performed by baseline age and RFF (±0.5 s) as sensitivity analysis.•Over up to 3.4 years, a difference in favour of drisapersen was seen for 6MWD and RFF.
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关键词
Duchenne muscular dystrophy,Antisense oligonucleotide,Comparative study,Exercise test,Dystrophin synthesis,Drug therapy
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