Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial
Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio M. Mercuri et autres
INTRODUCTION: vector genomes/kg) in ambulatory male patients with DMD aged 4 to < 8 years; N = 125. One-year results demonstrated the manageable safety of delandistrogene moxeparvovec, consistent with previous clinical trials. The primary endpoint (change from baseline in North Star Ambulatory …
us, gb, it, jp, es, de, ch (code pays fourni par la source)
5 citations
Neurology and Therapy