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Showing articles 0 to 2 of 2 Filter Applied: Werdnig-Hoffman disease (Click to remove) Risdiplam in Presymptomatic Spinal Muscular Atrophy NEJM 393:671-682, Finkel,R.S.,et al, 2025 Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017 Showing articles 0 to 2 of 2