SMA Treatments Have Completely Transformed: Here's What's New
Spinal Muscular Atrophy treatment has changed dramatically from an era when care focused almost entirely on managing symptoms to one where multiple therapies can target different parts of the disease. Spinraza and Evrysdi increase functional SMN protein through the backup SMN2 gene, while gene replacement therapies have expanded significantly. In November 2025, the FDA approved Itvisma, a one-time intrathecal gene therapy for adults and children 2 years of age and older with a confirmed SMN1 mutation. Unlike Zolgensma, which is administered intravenously to eligible children younger than 2, Itvisma delivers gene therapy directly into the cerebrospinal fluid and greatly expands the age range of people who may qualify for an SMA gene replacement treatment. These advances demonstrate how SMA treatment is moving beyond a single approach toward multiple strategies designed to protect motor neurons, increase SMN protein, and preserve physical function.
The transformation accelerated even further in 2026. On March 30, the FDA approved a high-dose Spinraza regimen, giving pediatric and adult SMA patients another nusinersen dosing option consisting of 50 mg loading doses and 28 mg maintenance doses. The approval was supported by the DEVOTE clinical program and represents the first major expansion of Spinraza dosing since the original treatment transformed SMA care in 2016. Then, on September 11, 2026, the FDA approved Isembyld, or apitegromab-mstn, for adults and children 2 years and older who are already receiving an SMN2-targeted therapy. Isembyld became the first FDA-approved SMA treatment specifically designed to directly target muscle loss, creating a new treatment strategy in which the disease can be addressed from both the motor-neuron and muscle sides. Even Evrysdi has continued evolving, with an FDA-approved tablet formulation joining the oral solution and giving eligible patients another way to take daily risdiplam.
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