DMD: other trials

Studies of progression of the disease:

  • NatHis-DMD: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
  • GNT-014-MDYF: Natural History of Duchenne Muscular Dystrophy – A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years
  • SRP4658-407: A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice

Database:

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

 

Acronym

NatHis-DMD

GNT-014-MDYF

SRP4658-407

UMD-DMD

Study Type RBM-HPS RBM-HPS Nat His Non-interventional data collection
Trial type International multicentre trial
Principal investigator Silvana De Lucia Silvana De Lucia Odile Boespflug-Tanguy Rabah Ben Yaou / France Leturcq / Sylvie Tuffery-Giraud
Sponsor AIM Gebethon SAREPTA French  dystrophinopathies molecular diagnosis laboratories + all NMD reference and competence centres
Fundings AIM Genethon SAREPTA
Study status Ongoing Ongoing In preparation  Ongoing
Recruitment status Closed Ongoing  Ongoing
Population Child Child Child  Adult / Child
Contact  r.benyaou@institut-myologie.org
+ infos on clinicaltrials.gov + infos on umd.be