3 results
- 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…
Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…
The primary aim of the trial is to demonstrate the non-inferiority of AMT-061 (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (AMT-061) follow-up compared to standard of…