A Phase 2, multicenter, single-arm study to evaluate the safety and dystrophin expression after Fordadistrogene Movaparvovec (PF-06939926) administration in male participants with early stage Duchenne Muscular Dystrophy
This is a gene therapy study that studies the safety and effectiveness of Fordadistrogene Movaparvovec (PF-06939926) in boys with DMD. It is a single-arm, non-randomized, open-label study, meaning that all participants will be receiving the gene therapy drug.
A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively. Part 1 is double-blind and randomised; Part 2 is open-label.
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 IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
This Phase 2b study is designed to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of vamorolone in comparison to corticosteroids and placebo treatments over a 24 week period. The study will also evaluate the persistence of the effect of vamorolone over a period of 48 weeks.
The study is designed to compare 2 different doses of Vamorolone to a standard dose of corticosteroids (prednisone at 0.75 mg/kg/day) and to a placebo. Across all sites, this trial aimed to recruit a total of 120 ambulant DMD patients ages 4 to <7 years.
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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