2 results
Approved WMOCompleted
The main objective is to determine the efficacy of Lyumjev in the Inreda AP system. Secondary objectives are: to assess safety, differences in pharmacodynamics and differences in AP-related outcome measures.
Approved WMOCompleted
Objectives for both the Treatment Period and Extension Period:Primary Objectives: - To assess safety and tolerability of the study medicine in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)- To assess the efficacy…