Study on Anifrolumab for Adults with Polymyositis or Dermatomyositis
The NetherlandsLong‑Term Safety and Efficacy Study of ENTR‑601‑45 and ENTR‑601‑44 in Participants with Duchenne Muscular Dystrophy
The NetherlandsSafety, Tolerability, Pharmacokinetics and Pharmacodynamics of DNL952 in Adults with Late‑Onset Pompe Disease
The NetherlandsDuchenne Muscular Dystrophy in Patients with Gene Mutations Amenable to Exon 44 Skipping Treated with Delpacibart Zotadirsen
The NetherlandsA study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1
The NetherlandsA study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy
The NetherlandsA study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.
The NetherlandsUsing 68Ga-FAPI-46 imaging to detect fibrosis in patients with inflammatory diseases such as Crohn's disease, ulcerative colitis, systemic sclerosis, and lung fibrosis.
The NetherlandsA Study of S-606001 Added to Enzyme Replacement Therapy for Patients with Late-Onset Pompe Disease
The NetherlandsA study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease
The NetherlandsA Study Testing AOC 1020 to Improve Movement in Adults with Facioscapulohumeral Muscular Dystrophy
The NetherlandsStudy of intravenous AOC 1001 to evaluate long-term safety and effectiveness in patients with myotonic dystrophy type 1
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