Astellas: A Randomized, Double-Blind, Placebo-Controlled Phase 1b Study with Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients with Duchenne Muscular Dystrophy (DMD)
To evaluate the safety and tolerability of ASP0367 as well as its efficacy on muscle function.
Ages 8-16 year old males with genetic confirmation of DMD
Sarepta Therapeutics Registry: A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.’s Exon-Skipping Therapies in Patients with Duchenne Muscular Dystrophy under Conditions of Routine Clinical Practice
To describe the usage, and potential benefits and risks of long-term use of eteplirsen or golodirsen in patients with Duchenne muscular dystrophy in routine clinical practice
No age restriction. Must be receiving eteplirsen or golodirsen medication or plan to start the medication within 6 months of enrollment.
Dr. Peter Karachunski: Imaging in DMD
To investigate dystrophic changes of muscle involvement in patients with DMD using imaging techniques such as MRI and ultrasound
To validate use of ultrasound for screening and monitoring purposes in patients with DMD during clinical studies and trials
Ages 10-30 year old males with genetic confirmation of DMD