DMD: other trials

Studies of progression of the disease:

  • NatHis-DMD: Outcome measures in Duchenne Muscular Dystrophy: a Natural History Study
  • Pre-U7: Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping (pre U7-53)

Database:

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

 

Acronym

NatHis-DMD

Pre-U7

UMD-DMD

Study Type RBM-HPS RBM-HPS Non-interventional data collection
Trial type International multicentre trial International multicentre trial
Principal investigator Laurent Servais Laurent Servais Rabah Ben Yaou / France Leturcq / Sylvie Tuffery-Giraud
Sponsor AIM Genethon / AIM French  dystrophinopathies molecular diagnosis laboratories + all NMD reference and competence centres
Fundings AIM Genethon
Study status Ongoing Ongoing  Ongoing
Recruitment status Closed Closed  Ongoing
Population Child Child  Adult / Child
Contact l.servais@institut-myologie.org l.servais@institut-myologie.org  r.benyaou@institut-myologie.org
+ infos on clinicaltrials.gov + infos on clinicaltrials.gov + infos on umd.be