Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

A. Strauss, Kevin; A. Farrar, Michelle; Muntoni, Francesco; Saito, Kayoko; R. Mendell, Jerry; Servais, Laurent; J. McMillan, Hugh; S. Finkel, Richard; J. Swoboda, Kathryn; M. Kwon, Jennifer; M. Zaidman, Craig; A. Chiriboga, Claudia; T. Iannaccone, Susan; M. Krueger, Jena; A. Parsons, Julie; B. Shieh, Perry; Kavanagh, Sarah; Tauscher-Wisniewski, Sitra; E. McGill, Bryan; A. Macek, Thomas

doi:10.1038/s41591-022-01866-4

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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial

Shared by NobleBlocks on Jun 17, 2022 • 12:00 AM UTC

Authors:

Kevin A. Strauss

Michelle A. Farrar

Francesco Muntoni

Abstract

Abstract SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SM...

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