Research for DMD

  • 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