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 WMORecruiting
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…