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Ventilation used overnight or during daytime naps is acceptable.","ALL","5 Years","17 Years",{"count":349,"type":350},60,"ESTIMATED","INTERVENTIONAL",[353],"PHASE2","This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).",[356],"Spinal Muscular Atrophy (SMA)",[358,359,360,361,362,363],"Spinal Muscular Atrophy","Pediatric","Muscle Function","Fatigability","SMA","ambulant","2026-05-20",{"date":366,"type":367},"2026-05-22","ACTUAL",{"date":369,"type":367},"2025-12-19",{"date":371,"type":350},"2029-05-28",{"name":5,"class":6},17]