2 results
Approved WMOCompleted
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…
Approved WMOCompleted
Primary objective: • To assess the pharmacokinetics of DOR in patients prior to and after BSSecondary objectives: • To assess the safety of DOR in patients prior to and after undergoing BS• To assess the viral response to DOR in patients prior to…