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Zolgensma (onasemnogene abeparvovec-xioi) Treatment for Spinal Muscular Atrophy

Zolgensma is a gene therapy approved by the US Federal Drug Administration (FDA) for children less than 2 years of age with spinal muscular atrophy (SMA). Children’s of Alabama began SMA treatments with Zolgensma in August 2019.

Zolgensma frequently asked questions:

Is my child eligible for Zolgensma treatment? According to hospital protocol and FDA regulation, your child must have the survival motor neuron 1 (SMN1) genetic mutations that causes SMA. Prior to starting therapy, this much be confirmed with genetic testing results.

According to the FDA, patients of all ages with all types of SMA less than 2 years of age. At Children’s of Alabama we are currently dosing pediatric patients and accepting new referrals.

How is Zolgensma given to patients? Zolgensma is given as a one-time, intravenous (IV) infusion that takes approximately 60 minutes. At Children’s of Alabama, patients receive this therapy in the outpatient infusion clinic.

Will my child’s insurance cover Zolgensma treatment? In Alabama, insurance coverage varies based on your plan. We recommend talking to your insurance provider as well as our medical team to determine the best course of action. We work with each insurance provider and family to obtain prior authorization before Zolgensma can be administered. Along with the Zolgensma medication dose, patients and families could incur costs from routine assessment, laboratory testing, and infusion clinic appointment. Children’s of Alabama is not currently accepting out-of-state Medicaid coverage for Zolgensma patients.

How do I get this treatment started for my child? All patients interested in Zolgensma must have established and ongoing care here at Children’s of Alabama. Our team will work with each family through the prior authorization process prior to scheduling your child for their infusion. For questions or interest regarding Zolgensma please call our MDA Care Center Coordinator, Shelley Coskery, at 205-638-2007.

For additional information, please visit the Avexis official website Zolgensma.com.