biostudies-literatureUnknown11(2)Servais L<h4>Background</h4>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.<h4>Objective</h4>We sought to describe patients with SMA treated with onasemnogene abeparvovec monotherapy in the real-world setting.<h4>Methods</h4>RESTORE is a prospective, multicenter, multinational, observational registry that captures data from a variety of sources.<h4>Results</h4>Recruitment started in September 2018. As of May 23, 2022, data were available for 168 patients treated with onasemnogene abeparvovec monotherapy. Median (IQR) age at initial SMA diagnosis was 1 (0-6) month and at onasemnogenJournal of neuromuscular diseases425-442https://www.ebi.ac.uk/biostudies/studies/S-EPMC10977451biostudies-literatureReal-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.PMC10977451Mercuri EProud CMDay JWFaulkner ESaito KRaju DLaMarca NDe Vivo DCAnderson FABenguerba KMMuntoni FFinkel RSTizzano EFShieh PBServais LSun RDesguerre IKirschner JQuijano-Roy SfalseReal-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.<h4>Background</h4>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.<h4>Objective</h4>We sought to describe patients with SMA treated with onasemnogene abeparvovec monotherapy in the real-world setting.<h4>Methods</h4>RESTORE is a prospective, multicenter, multinational, observational registry that captures data from a variety of sources.<h4>Results</h4>Recruitment started in September 2018. As of May 23, 2022, data were available for 168 patients treated with onasemnogene abeparvovec monotherapy. Median (IQR) age at initial SMA diagnosis was 1 (0-6) month and at onasemnogen2024-01-01T00:00:00Z20242026-07-16T04:12:35.977Z2025-04-05T23:17:08.569ZS-EPMC109774513825078310.3233/JND-230122