Whether you are just exploring your treatment options or have finished treatment with ZOLGENSMA® (onasemnogene abeparvovec-xioi), the resources below can help wherever your family is in their spinal muscular atrophy (SMA) journey.
If you receive a prenatal diagnosis, learn what you can do to prepare for your child to get treatment as soon as possible.
A helpful resource for preparing to talk to your child’s doctor about your treatment goals:
Step-by-step instructions with a checklist, starting from the first day of your ZOLGENSMA prescription to after infusion day:
A comprehensive guide about SMA and ZOLGENSMA, including:
or the Muscular Dystrophy Association (MDA).
How to talk to others about your child’s treatment—and a diagnosis they most likely haven’t heard of:
Your medical care team is there to help support you and your child every step of the way. In addition to your doctor, your team might include a lung specialist (pulmonologist) and a physical therapist.
Learn about medical care team members and other support for children with SMA with the Preparing for SMA after treatment PDF.
Temporarily, small amounts of ZOLGENSMA may be found in the patient's stool. Use good hand hygiene when coming into direct contact with patient body waste for one month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.
The safety information provided here is not comprehensive. Talk to the patient's doctor about any side effects that bother the patient or that don't go away.
You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 1-833-828-3947.
Please see the FullPrescribing Information.
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with patient body waste for one month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.
The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.
You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 1-833-828-3947.
Please see the Full Prescribing Information.
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The information provided in this site is intended only for audiences of the United States. This information does not take the place of talking to your health care professional about medical conditions and treatments. If you have questions about ZOLGENSMA after reading this information, ask your health care professional.
