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Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

key information

study id #: NCT03179631

condition: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn

status: recruiting

purpose:

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

intervention: Ataluren, PLACEBO

mechanism of action: Stop codon read through to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03179631

last updated: June 25, 2020

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