2 results
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…
Approved WMOCompleted
To assess the effectiveness and cost-effectiveness of personalized orthotic care to improve functioning in patients with NMD compared to standard orthotic care.