Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study

Title
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
Authors
Keywords
Spinal muscular atrophy, Clinical trial, Neurofilament, Newborn screening, Nusinersen, Presymptomatic
Journal
NEUROMUSCULAR DISORDERS
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2019-09-13
DOI
10.1016/j.nmd.2019.09.007

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