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Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

key information

study id #: NCT02369731

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in routine clinical practice.

intervention: Translarna

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT02369731

last updated: December 06, 2019

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