Sandra Reyna

Real-World Treatment Patterns in Patients with Spinal Muscular Atrophy Receiving Multiple Disease-Modifying Therapies

Nusinersen (approved in 2016), onasemnogene abeparvovec (intravenous infusion [OAV], 2019; intrathecal injection, 2025), and risdiplam (2020) are disease-modifying therapies (DMTs) available in the US for spinal muscular atrophy (SMA). Real-world evidence on treatment patterns am...