DMD Trials
Location Trial Status:
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 3 study that investigates the safety and efficacy of Viltolarsen in participants aged 4 to less than (<) 8 years old. The study has two groups: one group will take Viltolarsen, which skips Exon 53 out-of-frame mutations, and the other group will take a placebo. All participants will have weekly intravenous (IV) infusions of 80mg/kg Viltolarsen or placebo for 48 weeks. At the end of the study, participants may be asked to enrol in an open-label extension study, in which all participants will be taking Viltolarsen.
A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression.
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