3 results
1. What is the effect of a clinical oral dose of acetazolamide on the isocapnic hypoxic response (IHR), i.e. an exposure to hypoxia (end-tidal PO2 ~ 5.8 kPa) during 30 min? Does the agent increase the IHR as would be expected from the state of…
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…