Study on Anifrolumab for Adults with Polymyositis or Dermatomyositis
AustriaLong‑Term Safety and Efficacy Study of ENTR‑601‑45 and ENTR‑601‑44 in Participants with Duchenne Muscular Dystrophy
BelgiumSafety, 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
BelgiumA study to evaluate the efficacy and safety of DYNE-101 in patients with Myotonic Dystrophy Type 1
BelgiumA study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy
FranceA study to evaluate the long-term safety and effectiveness of BMN 351 in patients with Duchenne muscular dystrophy amenable to exon 51 skipping.
ItalyUsing 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
BelgiumA study testing the safety and effects of ADS-019 in adults aged 18 to 65 years with type 1 myotonic dystrophy
BelgiumA study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease
DenmarkA Study of Idecabtagene Vicleucel CAR-T Cell Therapy for Patients with Autoimmune Diseases That Did Not Respond to B Cell Removal Treatment
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