Duchenne Muscular Dystrophy

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

• GNT-016-MDYF: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory DMD
• MIS51ON : A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with DMD With Deletion Mutations Amenable to Exon 51 Skipping
• 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

Study of progression of the disease

• GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years

Database

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