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

C. De Vivo, Darryl; Bertini, Enrico; J. Swoboda, Kathryn; Hwu, Wuh‐Liang; O. Crawford, Thomas; S. Finkel, Richard; Kirschner, Janbernd; L. Kuntz, Nancy; Parsons, Julie; M. Ryan, Monique; J. Butterfield, Russell; Topaloğlu, Haluk; Ben‐Omran, Tawfeg; Sansone, Valeria; Jong, Yuh‐Jyh; Shu, Francy; F. Staropoli, John; A. Kerr, Douglas; Sandrock, Alfred; Stebbins, Christopher; Petrillo, Marco; Braley, Gabriel; Johnson, Kristina; Foster, Richard; Gheuens, Sarah; Bhan, Ishir; P. Reyna, Sandra; Fradette, Stephanie; Farwell, Wildon

doi:10.1016/j.nmd.2019.09.007

Public

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

Published in Neuromuscular Disorders • Sep 12, 2019

Authors:

Darryl C. De Vivo

Enrico Bertini

Kathryn J. Swoboda

Abstract

Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety outcomes as of March 29, 2019 in 25 children with genetically diagnosed SMA who first received nusinersen in infancy while pre...

Subject

Interim

Spinal muscular atrophy

Nature versus nurture

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