Frequently asked questions
Eli has 2 copies of SMN2 gene
Treated at ~4 months old
After receiving treatment with ZOLGENSMA,
Eli received another SMA treatment.
Want to learn more?
Below are some common questions about ZOLGENSMA and the support services available. For medical questions, your child’s doctor should always be your primary resource for information.
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 can halt spinal muscular atrophy (SMA) progression.
ZOLGENSMA is a gene replacement 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
SMA, or spinal muscular atrophy, is a progressive, rare genetic disease that affects the motor nerve cells in the spinal cord and impacts the muscles used for breathing, eating, crawling, and walking.
SMA is caused by the survival motor neuron 1 (SMN1) gene, which is missing or not working properly. This gene produces the survival motor neuron (SMN) protein that is critical to the nerves that control our muscles.
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.
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.
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, and complete blood count (including hemoglobin and platelet count).
Through Novartis Patient Supportᵀᴹ, 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.
Neuromuscular experts recommend early treatment for most 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 4 trials carefully to learn about the potential risks and potential benefits of ZOLGENSMA.
ZOLGENSMA is designed to target the genetic root cause of SMA with a one-time dose by replacing 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.
The most common side effects that occurred in children treated with ZOLGENSMA were elevated liver enzymes and vomiting. Read about ZOLGENSMA safety information.
Gene replacement 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 4 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.