3 results
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 main objective is to establish the feasibility of 166Ho-microspheres for intra-tumoural injections in OSCC by defining the total amount of leakage. The secondary objectives are to assess the safety profile by recording the adverse events, and…
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…