Feature|Articles|August 28, 2026

What’s Next in Spinal Muscular Atrophy: Pipeline Overview of Investigational Therapeutics

Author(s)Mariane Hernandez, Isabella Ciccone, MPH

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive® reviews the investigational agents currently moving through clinical development for spinal muscular atrophy.

Spinal muscular atrophy (SMA) is no longer therapeutically unaddressed. Nusinersen (Spinraza; Biogen), onasemnogene abeparvovec (Zolgensma; Novartis), and risdiplam (Evrysdi; Genentech/Roche) have been available for years, and the landscape expanded twice in the past 10 months: the FDA approved intrathecal onasemnogene abeparvovec (Itvisma; Novartis) for patients 2 years and older in November 2025, extending gene replacement beyond infancy, then cleared a high-dose nusinersen regimen in March 2026.^1,2