3 results
The study aims to limit the use of anthracyclines and to reduce the dose of ATRA. Another aim is to stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC >=10 x 109/l. Furthermore this study aims to…
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…