Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1
GermanyStudy on Low-Dose Aldesleukin for Patients with Pemphigus, Mucous Membrane Pemphigoid, Polymyositis, Dermatomyositis, and Primary Sclerosing Cholangitis
GermanyStudy on the Effectiveness of Dazukibart in Adults with Active Dermatomyositis or Polymyositis
GermanyStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
GermanyEfficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy
GermanyStudy on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease
GermanyLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
GermanyPhase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy
GermanyA study to evaluate the safety and effectiveness of CABA-201, fludarabine, and cyclophosphamide in patients with active inflammatory myopathy
GermanyStudy on the Effects of Rapcabtagene Autoleucel and Drug Combination for Patients with Severe Refractory Inflammatory Muscle Diseases
GermanyStudy on the Safety and Effectiveness of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy
GermanyStudy on the Effects of Human Normal Immunoglobulin in Adults with Dermatomyositis
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