Hopital d'Enfants CHU Timone

Marseille 13385

0 studies enrolling now · 2 studies all time

What they study most

Becker Muscular Dystrophy, Duchenne Muscular Dystrophy, GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease

Studies at this site

• GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) — Completed
• Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) — Stopped early