Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: 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; Parsons, Julie; B. Shieh, Perry; Kavanagh, Sarah; Wigderson, Melissa; Tauscher‐Wisniewski, Sitra; E. McGill, Bryan; A. Macek, Thomas

doi:10.1038/s41591-022-01867-3

Public

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

Published in Nature Medicine • Jun 17, 2022

Authors:

Kevin A. Strauss

Michelle A. Farrar

Francesco Muntoni

Abstract

Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutatio...

Subject

Spinal muscular atrophy

Medicine

Atrophy

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