2 results
Not approvedWill not start
To induce a vaccine specific immune response, hereby we hope to introduce the possibility of a new treatment to improve patient outcome and survival.
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…