French Muscular Dystrophy Association (AFM)

 

 

2014

• Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08) (website)

2013

• MYOMET: A randomized, double blind, placebo-controlled phase II study of metformin in myotonic dystrophy type 1 patients – FR (website)
• A phase I/II, non randomized, multicenter, open-label study of G1XCGD (lentiviral vector transduced CD34+ cells) in patients with X-Linked Chronic Granulomatous Disease (website)

2012

A Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy (website)