Study on Long-Term Safety and Efficacy of VX-670 for Adults with Myotonic Dystrophy Type I
GermanyStudy on the Safety of CNTY-101 and Aldesleukin for Patients with Active Autoimmune Diseases: Lupus, Scleroderma, and Myositis
GermanyStudy of ENTR-601-44 in Children with Duchenne Muscular Dystrophy Who Are Eligible for Exon 44 Skipping Treatment
GermanyStudy Comparing the Safety and Effectiveness of Mexiletine PR and Mexiletine IR for Adults with Non-Dystrophic Myotonias
GermanyStudy of ADS-010 for Adults with Facioscapulohumeral Muscular Dystrophy Type 1
GermanyStudy on the Safety of MB-CART19.1 for Patients with Active Systemic Lupus Erythematosus, Systemic Sclerosis, or Dermatomyositis/Polymyositis
GermanyStudy on the Effects of Mexiletine for Treating Myotonic Dystrophy Types 1 and 2 in Patients
GermanyLong-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat
GermanyStudy on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)
GermanyStudy on the Safety and Effects of SPK-3006 for Adults with Late-Onset Pompe Disease
GermanyLong-Term Safety and Efficacy Study of Mexiletine Hydrochloride for Patients with Myotonic Dystrophy Type 1 and Type 2
GermanyLong-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001
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