Study on Filgotinib for Patients with Refractory Behcet's Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
The NetherlandsStudy on the Safety and Efficacy of Avalglucosidase Alfa for Patients Aged 5 and Older with Non-Classic Pompe Disease
The NetherlandsLong‑term study of Pompe disease in all patients receiving alglucosidase alfa drug combination (miglustat, cipaglucosidase alfa, avalglucosidase alfa)
The NetherlandsLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
The NetherlandsPhase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy
The NetherlandsStudy on the Effects of Alglucosidase Alfa Enzyme Therapy in Children and Adults with Pompe Disease
The NetherlandsStudy on the Effects of Rapcabtagene Autoleucel and Drug Combination for Patients with Severe Refractory Inflammatory Muscle Diseases
The NetherlandsStudy on the Safety and Effectiveness of Filgotinib for Patients with Refractory Behcet's Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
The NetherlandsA study testing brepocitinib compared to placebo for adults with dermatomyositis to see if it works and is safe
The NetherlandsStudy of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy
The NetherlandsStudy on the Effectiveness and Safety of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
The NetherlandsStudy on AOC 1001 for Treating Myotonic Dystrophy Type 1 in Patients
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