Study on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1
BelgiumStudy on Low-Dose Aldesleukin for Patients with Pemphigus, Mucous Membrane Pemphigoid, Polymyositis, Dermatomyositis, and Primary Sclerosing Cholangitis
GermanyStudy on the Effects of Metformin for Improving Muscle Function in Patients with Myotonic Dystrophy Type 1
FranceStudy on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)
FranceStudy of doxecitine and doxribtimine oral solution for adult patients with Thymidine Kinase 2 (TK2) Deficiency
SpainStudy on the Effectiveness of Dazukibart in Adults with Active Dermatomyositis or Polymyositis
BelgiumStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
DenmarkEfficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy
BelgiumStudy on Filgotinib for Patients with Refractory Behcet's Disease, Idiopathic Inflammatory Myopathies, and IgG4-Related Disease
The NetherlandsStudy on the Effects of Avalglucosidase Alfa Enzyme Replacement Therapy in Patients with Pompe Disease
DenmarkStudy on Gene Therapy with AAV9.LAMP2B for Male Patients with Danon Disease
GermanyStudy on the Safety and Effects of SQY51 for Children and Adults with Duchenne Muscular Dystrophy
Francepowered by
