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A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From >=6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
study id #: NCT04336826
condition: Nonsene Mutation Duchenne Muscular Dystrophy
status: not yet recruiting
purpose:This study is designed to evaluate safety, tolerability, physical and motor development, and pharmacokinetics (PK) in children aged >=6 months to <2 years treated daily for 52 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
intervention: Ataluren
mechanism of action: Stop codon read through to promote dystrophin production
results: https://clinicaltrials.gov/ct2/show/results/NCT04336826?view=results
last updated: August 26, 2020
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