Duchenne Muscular Dystrophy (DMD) is an X-linked progressive muscular disorder caused by mutations in the dystrophin gene, affecting up to 1 in 5000 boys. Mutations within this gene result in a lack of expression of the functional form of its major protein product, dystrophin, causing damage to the muscle fibers, resulting in progressive muscle weakness and atrophy. The latter is responsible for the clinical manifestations of decreased physical functionality, respiratory and cardiac failure, and eventually death.
A Study to Evaluate the Efficacy and Safety of Casimersen and Golodirsen in patients with Duchenne Muscular Dystrophy (Sarepta Therapeutics)
Principal Investigator: Prof. Nathalie Goemans/ Prof. Liesbeth De Waele
Essence-Sarepta 301: 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.
Essence-Sarepta 302: Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen.
A study to Evaluate the Efficacy and Safety of Givinostat in patients with Duchenne Muscular Dystrophy (Italfarmaco)
Principal Investigator: Prof. Nathalie Goemans/ Prof. Liesbeth De Waele
Italfarmaco 48: Randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of Givinostat in ambulant patients with Duchenne Muscular Dystrophy.
Italfarmaco 51: Open-label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies.
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study to Assess the Efficacy and Safety of Vamorolone in Ambulatory Boys with Duchenne Muscular Dystrophy (ReveraGen)
Principal Investigator: Prof. Nathalie Goemans/ Prof. Liesbeth De Waele