Frequently asked questions
Treated at ~4 months old
Frequently asked questions
Want to learn more?
Below are some common questions about ZOLGENSMA® (onasemnogene abeparvovec-xioi) and the support services available. For medical questions, your doctor should always be your primary resource for information.
Can I wait to treat SMA?
The sooner treatment with ZOLGENSMA is given, the sooner muscle function may be preserved. For children with 2, 3, or 4 SMN2 backup gene copies, early treatment with ZOLGENSMA is recommended to halt spinal muscular atrophy (SMA) progression.
What is ZOLGENSMA?
ZOLGENSMA is a gene therapy approved by the US Food and Drug Administration (FDA) for children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is:
- A one-time-only dose
- Given intravenously (IV)
- An infusion that takes 60 minutes
How is ZOLGENSMA given?
ZOLGENSMA is given intravenously as a one-time infusion. The actual infusion takes about 60 minutes. Learn more about the step-by-step process of receiving ZOLGENSMA.
What if my child’s adeno-associated virus 9 (AAV9) antibody levels are too high to receive ZOLGENSMA?
Talk to your doctor about monitoring this antibody level. If your child’s immune system has built up a certain level of anti-AAV9 antibodies, they might not be eligible to receive ZOLGENSMA right away.
Ask your child’s doctor to continue monitoring your child’s AAV9 antibody levels—they can decrease over time and your child may become eligible to receive ZOLGENSMA.
How will my child’s doctor follow up after a ZOLGENSMA infusion?
You and your child’s doctor will schedule a number of follow-up appointments over the next 3 months or longer. Your child’s doctor will be carefully looking at liver function, as well as other blood work and clinical exams.
Your child’s monitoring schedule over 3 months:
All results are compared against a baseline assessment taken before your child’s infusion recording liver function, creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I.
Can I get help with insurance?
Through our OneGene Program®, families are assigned a Case Coordinator who acts as a go-between to connect you to resources you may need and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible children.
Does ZOLGENSMA halt SMA progression?
Early treatment is recommended for all children with SMA. Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment. Please read the results of the 3 trials carefully to learn about the potential risks and potential benefits of ZOLGENSMA.
How does gene therapy work?
ZOLGENSMA is designed to target the genetic root cause of SMA with a one-time dose by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene. The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions. Read more about how ZOLGENSMA works.
What are the possible or likely side effects of ZOLGENSMA?
The most common side effects that occurred in children treated with ZOLGENSMA were elevated liver enzymes and vomiting.
Read about ZOLGENSMA safety information.
How long do the benefits from ZOLGENSMA last?
Gene therapy in SMA is designed to keep producing SMN protein in a child’s body, helping to preserve muscle function.
Please read the results of the 3 trials carefully to learn about the risks and benefits of ZOLGENSMA. Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.