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

U.S., Feb. 12 -- ClinicalTrials.gov registry received information related to the study (NCT07403214) titled 'A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy' on Feb. 04.

Brief Summary: 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.

Study Start Date: June 04, 2025

Study Type: OBSERVATIONAL

Condition: Muscular Atrophy, Spinal ...