Is there a treatment or a cure for SMA?
Disease-modifying drugs are available if eligibility criteria is met. Zolgensma (Onasemnogene Abeparvovec) is available for those with SMA type 1. Risdiplam (Evrysdi) and Nusinersen (Spinraza) are available for those with SMA types 1, 2, or 3. These drugs are most effective when given before muscle weakness onset. They can stop the progression of muscle weakness and may improve strength and motor function.
Information on Zolgensma (Onasemnogene Abeparvovec)