3 results
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…
The first main objective is to investigate the pharmacokinetic properties of dry powder amikacin at different dosages and compare the peak serum values to a single i.v. dose. The secondary main objective is to assess the local tolerability of dry…
-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…