Duchenne Muscular Dstrophy

Duchenne muscular dystrophy (DMD) affects the ensemble of the muscles of the organism: skeletal muscle, cardiac muscle, and some smooth muscle. The first symptoms appear in childhood at about 3 years of age. Only boys are affected. DMD is characterized by progressive muscle weakness of the limbs and the trunk. Going upstairs, then walking become difficult and then impossible. Respiratory and cardiac effects may be lethal.

Clinical trials on DMD ongoing at the Institute:

Pharmacological trials

  • WAVE: A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administrated Intravenously to Patients with Duchenne Muscular Dystrophy
  • WAVE-OLE: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
  • SRP4053: A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
  • Italfarmaco: Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
  • Essence: Study of SRP-4045 and SRP-4053 in DMD Patients
  • SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid steroids
  • PTC-PASS: Long-Term Observational Study of Translarna Esafety and effectiveness in usual care
  • Anti-Myostatin: A Phase 2/3, Randomized,Parallel, Double-blind, Placebo-controlled, to assess the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy
  • SRP 4658-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping

Studies of progression of the disease

  • NatHis-DM: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
  • Pre-U7: Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Database

  • UMD-DMD: Database Dystrophinopathies (UMD-DMD France) (www.umd.be/DMD)