Study on Anifrolumab for Adults with Polymyositis or Dermatomyositis
DenmarkA study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1
DenmarkA Study of S-606001 Added to Enzyme Replacement Therapy for Patients with Late-Onset Pompe Disease
DenmarkA study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease
DenmarkA Study Testing AOC 1020 to Improve Movement in Adults with Facioscapulohumeral Muscular Dystrophy
DenmarkStudy of intravenous AOC 1001 to evaluate long-term safety and effectiveness in patients with myotonic dystrophy type 1
DenmarkStudy on the Effects of Mexiletine for Treating Myotonic Dystrophy Types 1 and 2 in Patients
DenmarkStudy on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)
DenmarkLong-Term Safety and Efficacy Study of Mexiletine Hydrochloride for Patients with Myotonic Dystrophy Type 1 and Type 2
DenmarkStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
DenmarkStudy on the Effects of Avalglucosidase Alfa Enzyme Replacement Therapy in Patients with Pompe Disease
DenmarkLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
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