Treatments and Outcomes for Patients with Spinal Muscular Atrophy Type 2: Findings from RESTORE Registry (P7-9.010)

Servais, Laurent; Benguerba, Kamal; C. De Vivo, Darryl; Kirschner, Janbernd; Muntoni, Francesco; M. Proud, Crystal; F. Tizzano, Eduardo; Quijano‐Roy, Susana; Desguerre, Isabelle; Saito, Kyoko; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; Anderson, Fred; Faulkner, Eric; S. Finkel, Richard

doi:10.1212/wnl.0000000000202600

Public

Treatments and Outcomes for Patients with Spinal Muscular Atrophy Type 2: Findings from RESTORE Registry (P7-9.010)

Published in Neurology • Apr 25, 2023

Authors:

Laurent Servais

Kamal Benguerba

Darryl C. De Vivo

Abstract

We sought to describe real-world treatment patterns and outcomes for patients with SMA type 2 (SMA2) receiving onasemnogene abeparvovec (OA) monotherapy or who switched to OA from nusinersen.

Subject

Spinal muscular atrophy

Medicine

Atrophy

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