Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1
ItalyStudy on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)
ItalyStudy on the Effectiveness of Dazukibart in Adults with Active Dermatomyositis or Polymyositis
ItalyStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
ItalyEfficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy
ItalyStudy on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease
ItalySafety 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)
ItalyPhase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy
ItalyA study to evaluate the safety and effectiveness of CABA-201, fludarabine, and cyclophosphamide in patients with active inflammatory myopathy
ItalyStudy on the Effects of Rapcabtagene Autoleucel and Drug Combination for Patients with Severe Refractory Inflammatory Muscle Diseases
ItalyStudy on the Effectiveness of Deflazacort in Treating LMNA-related Congenital Muscular Dystrophy in Adults and Children
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