Treatment

GOLODIRSEN (SRP-4053) clinical trials

1 indexed studies · 0 currently recruiting

Ongoing, recruitingTherapeutic confirmatory (Phase III)
A Double-blind, Placebo-Controlled, Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy
This study is looking at two new medicines, SRP-4045 and SRP-4053, for Duchenne muscular dystrophy. It wants to see if they can help boys with certain types of Duchenne walk better and for longer. The study will compare these medicines to a dummy treatment.
For: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping.
Belgium · Ireland · Poland