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A Phase III Open-Label, Single-Group, Extension Study to
Obtain Long-Term Safety and Tolerability Data of
Idebenone in the Treatment of Friedreich*s Ataxia Patients

- To gather long-term data on the safety and tolerability of idebenone inFriedreich*s Ataxia- To explore the effect of idebenone after longer term administration onneurological symptoms and signs as assessed by the InternationalCooperative Ataxia…

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)

The primary objective of this study is to evaluate the long-term safety and tolerability of OV101 in individuals with AS assessed by the incidence and severity of AEs and SAEs in subjects who are at least 2 years old.The secondary objectives of this…

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

Primary* Determine efficacy by demonstrating achievement of developmental milestone of sitting without support up to 18 months of age as defined by WHO Motor Developmental Milestones[22].Secondary* Determine efficacy based on survival at 14 months…