Study on Long-Term Safety and Efficacy of VX-670 for Adults with Myotonic Dystrophy Type I
The NetherlandsStudy of ENTR-601-45 Safety and Effectiveness in Patients with Duchenne Muscular Dystrophy Who Can Skip Exon 45
The NetherlandsStudy of ADS-010 for Adults with Facioscapulohumeral Muscular Dystrophy Type 1
The NetherlandsLong-Term Safety and Efficacy Study of Givinostat for Patients with Duchenne Muscular Dystrophy Previously Treated with Givinostat
The NetherlandsStudy on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)
The NetherlandsStudy on the Safety and Effects of Givinostat in Young Children with Duchenne Muscular Dystrophy (Ages 2 to Less Than 6 Years)
The NetherlandsStudy 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 NetherlandsStudy on the Safety of VX-670 for Adults with Myotonic Dystrophy Type 1
The NetherlandsStudy on ATX-01 for Adults Aged 18-64 with Myotonic Dystrophy Type 1 (DM1)
The NetherlandsStudy on the Safety and Effects of Cipaglucosidase Alfa and Miglustat for Children with Infantile-onset Pompe Disease
The NetherlandsEfficacy and Safety of Givinostat for Non-Ambulant Patients with Duchenne Muscular Dystrophy
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