Understanding the Progression of Treatments for SMA

As research advanced, the SMA community began seeing disease-modifying treatments that target the biology behind the condition rather than only treating complications. There are currently four FDA-approved therapies for SMA: Spinraza, Zolgensma, Evrysdi, and Itvisma.

The treatment landscape for Spinal Muscular Atrophy changed significantly when Spinraza became the first FDA-approved therapy for SMA in 2016, followed by Zolgensma in 2019 and Evrysdi in 2020. Cure SMA describes Zolgensma as a gene therapy approved to treat children with all types of SMA under two years of age, while Evrysdi was approved for SMA in all ages and all types. More recently, the FDA approved Itvisma on November 24, 2025, for adult and pediatric patients two years of age and older with a confirmed SMN1 gene mutation. These milestones matter because they show how SMA treatment has progressed from supportive care alone to options that may help increase SMN protein or replace the missing or faulty SMN1 gene.

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