Risdiplam in Presymptomatic Spinal Muscular Atrophy
NEJM 393:671-682, Finkel,R.S.,et al, 2025
Idiopathic Normal-Pressure Hydrocephalus
NEJM 393:2243-2253, 2264, Johnson,M.D.,& Williams, M.A., 2025
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017