Moving forward after treatment

Once your child has been treated with ZOLGENSMA® (onasemnogene abeparvovec-xioi), there is still a need to manage your child’s spinal muscular atrophy (SMA). Learn more about additional accommodations or therapy, how to educate your family and friends about SMA, and ways to connect with advocacy group resources for support.

Download the SMA After Treatment guide

Moving forward after treatment

Once your child has been treated with ZOLGENSMA® (onasemnogene abeparvovec-xioi), there is still a need to manage your child’s spinal muscular atrophy (SMA). Learn more about additional accommodations or therapy, how to educate your family and friends about SMA, and ways to connect with advocacy group resources for support.

Download the SMA After Treatment guide

Ongoing management of SMA

ZOLGENSMA is not a cure and cannot reverse damage already caused by SMA before treatment. Your child may continue to show signs and symptoms of SMA now or in the future. These may include difficulty swallowing, difficulty breathing, or muscle weakness. Additional therapies, accommodations, and support may be needed to help manage your child’s SMA and guide his or her ongoing development, including:

  • A healthcare team personalized to your child’s needs, which usually includes a pediatric neurologist who serves as the coordinator of care
  • Breathing support, which may be needed because SMA affects the muscles used to breathe
  • Nutritional support, which may be needed because SMA can affect the ability to swallow and because proper nutrition is key to overall health and growth
  • Supportive therapy, which may be needed depending on your child’s muscle strength and may include physical therapy and positioning equipment, such as a wheelchair

Donovan, who has SMA Type 1, uses a cough assist machine in the morning to help with breathing. He was treated at ~2 months and is pictured at 2½ years.

Donovan, who has SMA Type 1, using a cough assist machine

Tenley, who has SMA Type 1, uses a BiPAP machine for breathing support at night. She was treated at ~5½ months and is pictured at 4½ years.

Tenley, who has SMA Type 1, using a BiPAP machine

Adalyne, who has SMA Type 1, performs stretches and wears custom braces to help her feet. She was treated at ~2½ months and is pictured at 3 years.

Adalyne, who has SMA Type 1, stretching with her mom
Download SMA After Treatment

SMA After Treatment

Learn more about additional therapies, accommodations, and support with the SMA After Treatment guide.

Download the guide

Educate family and friends

The more people learn about SMA and ZOLGENSMA, the more they can support families and help educate others. The Explaining SMA and ZOLGENSMA handout covers facts about SMA, how ZOLGENSMA works, how to find advocacy groups, and more.

Download the handout

Connect with others

Get additional support from advocacy organizations and other community groups that help families living with SMA.

See organizations

Support for caregivers

The Courageous Parents Network is created by parents, for parents, to support, guide, and strengthen families as they care for a seriously ill child. Through shared experiences, parents will find valuable information, helpful tools, and virtual support to help them at every step of their journey.

Visit Courageous Parents Network

We go to the SMA clinic once every 6 months to make sure we’re moving in the right direction. We see a pulmonologist, dietitian, neurologist, and physical therapist. Natalie, mother of Eli

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • Liver enzymes could become elevated and cause acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • Liver enzymes could become elevated and cause acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
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 the 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 AveXis at 833-828-3947.

Please see the Full Prescribing Information.