Study of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk
BelgiumStudy on the Safety and Efficacy of Avalglucosidase Alfa for Patients Aged 5 and Older with Non-Classic Pompe Disease
The NetherlandsLong‑term study of Pompe disease in all patients receiving alglucosidase alfa drug combination (miglustat, cipaglucosidase alfa, avalglucosidase alfa)
The NetherlandsSafety and dose‑finding study of CD19‑CART01 in patients with SLE, SSc, DM/PM, IMNM, AAV, MG, NMOSD, or chronic GVHD
ItalyLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
DenmarkPhase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy
BelgiumA study to evaluate the safety and effectiveness of CABA-201, fludarabine, and cyclophosphamide in patients with active inflammatory myopathy
GermanyStudy of ASP2957 Gene Therapy for Male Patients with X-linked Myotubular Myopathy Who Require Breathing Support with Ventilators
FranceStudy on the Effects of Alglucosidase Alfa Enzyme Therapy in Children and Adults with Pompe Disease
The NetherlandsStudy on the Safety and Effectiveness of DT-DEC01 for Patients with Duchenne Muscular Dystrophy
PolandStudy on the Effects of Rapcabtagene Autoleucel and Drug Combination for Patients with Severe Refractory Inflammatory Muscle Diseases
AustriaStudy on the Effectiveness of Deflazacort in Treating LMNA-related Congenital Muscular Dystrophy in Adults and Children
Italypowered by
