Clinical trial

A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

Novartis PharmaceuticalsCompletedStarted Jun 4, 20254805 enrolledUnited StatesNCT07403214

Abstract

The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world. The DMTs included onasemnogene abeparvovec-xioi (OA), nusinersen, and risdiplam. This study was conducted using data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.

Keywords

Spinal muscular atrophyMultiple disease modifying therapiesReal-world evidenceTreatment patternsMuscular Atrophy, Spinal

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