How is Zolgensma administered?
Zolgensma is given as a one-time, single dose intravenous (IV) infusion into a vein at a treatment center. The infusion takes approximately one hour. Your healthcare provider can direct you to the closest treatment center.
The dose for Zolgensma is based on the weight of the child and will be determined by your doctor.
The child will also receive an oral corticosteroid medication before and after Zolgensma treatment. Contact the doctor right away if the child misses a dose of the corticosteroid or vomits it up.
Regular blood tests are required to check on liver function. Contact your doctor immediately if the child's skin or the whites of the eyes look yellowish. This could signal there is a problem with the liver.
Before the patient can receive Zolgensma, the doctor will need to confirm with lab tests that the child qualifies for treatment. This involves a genetic test, an antibody test, and tests to monitor the health of the liver and the blood.
Zolgensma (onasemnogene abeparvovec-xioi) is approved to treat children less than 2 years of age with spinal muscular atrophy (SMA) mutations in the survival motor neuron 1 (SMN1) gene.
Zolgensma not a cure for spinal muscular atrophy (SMA) and does not reverse any damage already caused by SMA before treatment. It has not been studied in children with advanced SMA (for example, with complete paralysis of limbs or permanent ventilator dependence).
What is Spinal Muscular Atrophy (SMA)?
Spinal muscular atrophy (SMA) is a rare genetic motor neuron disease that leads to problems with breathing, holding up the head, and swallowing.
The majority of children with SMA do not survive past early childhood due to respiratory failure (inability to breathe). In its most severe form, SMA can lead to permanent ventilation or death by age two.
This is not all the information you need to know about Zolgensma (onasemnogene abeparvovec-xioi) for safe and effective use and does not take the place of talking to your doctor about your treatment. Review the full Zolgensma information here, and discuss this information and any questions you have with your doctor or other health care provider.
- Starting Zolgensma. Zolgensma.com. Accessed Oct. 7, 2021 at https://www.zolgensma.com/starting-zolgensma
- Zolgensma (onasemnogene abeparvovec-xioi) prescribing information. Novartis. 5/2021. Accessed Oct. 7, 2021 at https://www.novartis.us/sites/www.novartis.us/files/zolgensma.pdf
- A caregiver's guide to Zolgensma. Novartis Gene Therapies Inc. 3/2021. Bannockburn, IL. Accessed Oct. 7, 2021 at https://www.zolgensma.com/pdf/a-caregivers-guide-to-zolgensma.pdf
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- Zolgensma Information for Consumers
- Zolgensma Information for Healthcare Professionals (includes dosage details)
- Side Effects of Zolgensma (detailed)