Study on the Effects of Mexiletine for Treating Myotonic Dystrophy Types 1 and 2 in Patients
BelgiumLong-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat
BelgiumStudy on Baricitinib for Patients with Relapsing or Naïve Dermatomyositis
FranceStudy on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)
DenmarkStudy on the Safety and Effects of SPK-3006 for Adults with Late-Onset Pompe Disease
GermanyStudy on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)
BelgiumStudy on Ruxolitinib for Treating Inclusion Body Myositis in Patients
FranceLong-Term Safety and Efficacy Study of Mexiletine Hydrochloride for Patients with Myotonic Dystrophy Type 1 and Type 2
BelgiumStudy on Upadacitinib for Patients with Idiopathic Inflammatory Myopathies After Stopping IVIG
AustriaStudy on the Effect and Safety of Autologous Mesoangioblasts for Patients with Mitochondrial Myopathy Due to m.3243A>G Mutation
The NetherlandsStudy on Reducing Treatment Frequency of Alglucosidase Alfa for Elderly Patients with Late-Onset Pompe Disease
The NetherlandsLong-term Safety Study of Delandistrogene Moxeparvovec for Patients with Duchenne Muscular Dystrophy Who Previously Received SRP-9001
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