Real-World Use of Novel Treatments in Patients with Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

Principal Investigator: Dr. Ed Leung

The primary objective of this study is to describe demographics and baseline clinical characteristics of Spinal Muscular atrophy (SMA) pediatric patient treated with at least one of the three distinct disease modifying treatments (DMTs)(nusinersen, risdiplam, or onasemnogene ) or combinations of the three in various geographies outside of the United States (US).