Study on Anifrolumab for Adults with Polymyositis or Dermatomyositis
GermanySafety, Tolerability, Pharmacokinetics and Pharmacodynamics of DNL952 in Adults with Late‑Onset Pompe Disease
GermanyDuchenne Muscular Dystrophy in Patients with Gene Mutations Amenable to Exon 44 Skipping Treated with Delpacibart Zotadirsen
GermanyA study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1
GermanyA study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy
GermanyA Study of S-606001 Added to Enzyme Replacement Therapy for Patients with Late-Onset Pompe Disease
GermanyA study testing the safety and effects of ADS-019 in adults aged 18 to 65 years with type 1 myotonic dystrophy
GermanyA study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease
GermanyA Study of Idecabtagene Vicleucel CAR-T Cell Therapy for Patients with Autoimmune Diseases That Did Not Respond to B Cell Removal Treatment
GermanyA Study Testing AOC 1020 to Improve Movement in Adults with Facioscapulohumeral Muscular Dystrophy
GermanyStudy of AlloNK and rituximab with cyclophosphamide and fludarabine in adults with relapsing rheumatoid arthritis, inflammatory myopathies, systemic sclerosis, or Sjögren's disease
GermanyStudy of intravenous AOC 1001 to evaluate long-term safety and effectiveness in patients with myotonic dystrophy type 1
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