2 results
Approved WMOWill not start
PRIMARY• To determine the efficacy on study Day 28 of the IT-combination in inducing a clinical response in patients with severe acute GVHD refractory to first line therapy with intermediate dose corticosteroids. SECONDARY• To evaluate the overall…
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…