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

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 sought to describe patients with SMA treated with onasemnogene abeparvovec monotherapy in the real-world setting.

Methods

RESTORE is a prospective, multicenter, multinational, observational registry that captures data from a variety of sources.

Results

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 onasemnogen

SUBMITTER: Servais L 

PROVIDER: S-EPMC10977451 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature