Study on the Effectiveness and Safety of Avalglucosidase Alfa for Babies with Infantile-Onset Pompe Disease
BelgiumStudy of DYNE-251 safety and effectiveness in patients with Duchenne muscular dystrophy who are candidates for exon 51 skipping treatment
BelgiumStudy on the Effects of Fordadistrogene Movaparvovec for Patients with Duchenne Muscular Dystrophy
BelgiumStudy on Long-term Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Adults with Late-onset Pompe Disease
BelgiumStudy on the Effectiveness and Safety of Satralizumab for Patients with Type 1 Facioscapulohumeral Muscular Dystrophy
FranceStudy on the Safety and Efficacy of Cipaglucosidase Alfa and Miglustat for Children with Late-onset Pompe Disease
GermanyStudy on the Safety and Effectiveness of ABC008 for Patients with Inclusion Body Myositis
BelgiumGene Therapy Study for Patients with Limb-Girdle Muscular Dystrophy Using GNT0006
DenmarkStudy on the Long-term Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
BelgiumStudy on the Effects of EDG-5506 in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy
DenmarkStudy on the Effects of EDG-5506 for Adults and Adolescents with Becker Muscular Dystrophy
BelgiumStudy on the Effects of Enpatoran for Patients with Dermatomyositis and Polymyositis
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