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Study of Ataluren in >=2 to <5 Year-Old Males 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, and pharmacokinetics (PK) of ataluren in patients aged >=2 to <5 years old with Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene. The study includes a 4-week screening period, a 4-week study period, and a 48-week extension period for patients who complete the 4-week study period (52 weeks total treatment).

intervention: Ataluren

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

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

last updated: July 08, 2019