Study on the Effects and Safety of Efgartigimod PH20 SC for Adults with Active Idiopathic Inflammatory Myopathy
The NetherlandsStudy on Long-Term Safety and Effectiveness of Vamorolone in Boys with Duchenne Muscular Dystrophy Who Completed Previous Vamorolone Studies
The NetherlandsStudy on Eteplirsen for Patients with Duchenne Muscular Dystrophy with Deletion Mutations Amenable to Exon 51 Skipping
The NetherlandsStudy on the Effects of Ribitol (BBP-418) in Patients with Limb Girdle Muscular Dystrophy Type 2I
The NetherlandsStudy on the Effects of Pariglasgene Brecaparvovec for Adults with Glycogen Storage Disease Type Ia
The NetherlandsStudy on the Safety and Efficacy of Viltolarsen for Boys with Duchenne Muscular Dystrophy
The NetherlandsSafety and effectiveness study of DYNE-101 in patients with Myotonic Dystrophy Type 1 (DM1)
The NetherlandsStudy to Determine the Best Dose of Vesleteplirsen for Patients with Duchenne Muscular Dystrophy Suitable for Exon 51-Skipping Treatment
The NetherlandsStudy of Pariglasgene Brecaparvovec and Prednisolone for Adults with Glycogen Storage Disease Type Ia
The NetherlandsStudy on the Safety and Effects of mRNA-3745 for Patients with Glycogen Storage Disease Type 1a (GSD1a)
The NetherlandsStudy on the Safety and Effects of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1)
The NetherlandsStudy on the Effectiveness and Safety of Avalglucosidase Alfa for Babies with Infantile-Onset Pompe Disease
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