Treatments for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) can’t be cured. This makes early diagnosis of SMA important. Treatment may depend on your age, overall health and the type of SMA you have.

The FDA has approved two medications to treat SMA: nusinersen and onasemnogene abeparvovec-xioi. Both are forms of gene therapy that affect the SMN1 and SMN2 genes, which are involved in SMA. These genes give your body instructions for making a protein that helps control muscle movement.

Nusinersen is FDA-approved for both pediatric and adult patients with genetically confirmed SMA. This medication is given as an injection into the spinal canal. Northwestern Memorial Hospital is currently one of just two providers in Illinois offering treatment with nusinersen. We are treating a growing number of patients who have different types of SMAs, including types 3 and 4.

We also offer treatment with onasemnogene abeparvovec-xioi for pediatric patients. It is given as an intravenous (into the vein) infusion.

To qualify for the latest medication therapies, your insurance company may require genetic testing. Our team includes genetic counselors who offer this testing at Northwestern Memorial Hospital.