Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Servais, Laurent; W. Day, John; C. De Vivo, Darryl; Kirschner, Janbernd; Mercuri, Eugenio; Muntoni, Francesco; M. Proud, Crystal; B. Shieh, Perry; F. Tizzano, Eduardo; Quijano‐Roy, Susana; Desguerre, Isabelle; Saito, Kayoko; Faulkner, Eric; Benguerba, K.; Raju, Dheeraj; LaMarca, Nicole; Sun, Rui; A. Anderson, Frederick; S. Finkel, Richard

doi:10.3233/jnd-230122

Public

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Published in Journal of Neuromuscular Diseases • Jan 18, 2024

Authors:

Laurent Servais

John W. Day

Darryl C. De Vivo

Abstract

Background: Long-term, real-world effectiveness and safety data of disease-modifying treatments for spinal muscular atrophy (SMA) are important for assessing outcomes and providing information for a larger number and broader range of SMA patients than included in clinical trials. Objective: We sough...

Subject

Medicine

SMA*

Spinal muscular atrophy

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