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Study of Ataluren in >=2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

key information

study id #: NCT02819557

condition: Duchenne Muscular Dystrophy

status: completed


This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged >=2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

intervention: Ataluren

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

results: https://clinicaltrials.gov/ct2/show/results/NCT02819557

last updated: September 08, 2020