NARRATOR: Welcome to the steps to treatment for ZOLGENSMA® (onasemnogene abeparvovec-xioi).
Once you and your child’s doctor have chosen treatment with ZOLGENSMA, there are a few steps that need to be taken.
Before ZOLGENSMA can be given to your child, a diagnosis of spinal muscular atrophy, or SMA, has to be confirmed by a genetic test. Your doctor will order a genetic test for your child to confirm the SMN1 gene is missing or nonworking. In addition, your doctor may also test your child’s SMN2 gene copy number. Your doctor will also need to perform additional lab tests—an AAV9 antibody test and baseline medical tests. These tests include liver function, creatinine, complete blood count, including hemoglobin and platelet count, and troponin-I.
While waiting for the genetic confirmation and AAV9 antibody tests, your doctor can submit 2 forms: a ZOLGENSMA Prescription Form and a Patient Consent Form. By signing the consent form, you will be connected with the OneGene Program. This is a dedicated resource provided by Novartis Gene Therapies that supports your family and helps you to navigate the insurance process, including prior authorizations and benefits.
You will also be assigned a Patient Resource Manager, who can share educational resources and guidance about preparing for treatment.
Your doctor’s office will follow up on the status of your insurance coverage for ZOLGENSMA. If approved, and if not previously completed, your doctor will perform baseline medical tests before treatment with ZOLGENSMA.
Your doctor will also prescribe an oral corticosteroid, a medicine that must be given to your child the day before infusion, on infusion day, and continued after infusion as directed by your doctor. This will help manage potential reactions to ZOLGENSMA by your child’s immune system.
On treatment day your child will be infused with ZOLGENSMA. The actual ZOLGENSMA infusion takes about 60 minutes. Your child will also receive a second dose of the oral corticosteroid. After infusion, your doctor and care team will talk to you about post-treatment follow-up and additional medical monitoring.
You will continue to give your child the oral corticosteroid as it is prescribed by your doctor, which is recommended for at least 2 months after infusion. In addition to liver function, your doctor will monitor other markers through blood tests, which will require weekly appointments for the first month and less frequently in the second and third months, or longer, as determined by your doctor.
Contact your doctor immediately if your child misses a dose of the corticosteroid or vomits it up, or if their skin and/or whites of the eyes appear yellowish. You should also talk to your doctor about what to watch for after infusion, including the signs and symptoms of a viral respiratory infection, adjustments to your child’s vaccination schedule, and how to handle bodily waste in the first month.
Thrombotic microangiopathy (TMA) has been reported to occur approximately one week following infusion. Seek immediate medical attention if your child experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.
It’s important to understand that your child still has SMA after treatment with ZOLGENSMA. So work with your doctor and care team to understand any additional therapies and support that can help manage your child’s SMA. This could include physical and occupational therapy, along with nutritional and breathing support or cough assist, or both.
For more helpful information, read the complete steps to starting treatment at ZOLGENSMA.com.
If you have questions about ZOLGENSMA, talk to your doctor today.
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