ArticleLancet regional health. Americas2025

Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old from OFELIA, a phase 4, open-label, multicenter, non-randomised, interventional study.

Jonas Alex Saute et al.PubMed ↗Full text ↗Publisher ↗

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