4 results
Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To investigate the pharmacokinetics (PK) of risdiplam and metabolites as appropriate.Secondary ObjectiveThe secondary…
Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…
-Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA-Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points-Further evaluate…
The primary efficacy objective for this study is to demonstrate non-inferiority of ocrelizumab compared with fingolimod