Every Day With SMA

Navigating life with SMA

Scroll Down Icon

Every Day With SMA

Navigating life with SMA

Becoming the advocate for your child with SMA

Speak up and be heard

Hear from SMA families as they share their perspective on speaking up for their child even after treatment.
Read the transcript

NATALIE: I think early on as an SMA mom, other moms will teach you to advocate and fight for your child. I feel like when your child struggles on a daily basis with things, you’re gonna fight just a little bit harder.

MILAN: Several times we have gone to doctors and to pediatricians for Evelyn’s checkups or meet a new pediatrician and they have no idea what SMA is. It’s important to be able to communicate with people and explain who Evelyn is and where she’s come from and the struggles that she has.

ELENA: When we have to go to a doctor I am prepared to, to just give a perspective to the doctor. Ah, and help them to, to see how, ah, how they can help.

TINA: When you’re stepping into an atmosphere, where they’re not familiar with your child or your child’s case, you need to make sure that you’re being heard. Sometimes it’s very hard but us as parents, we know our children, and we know what works and what doesn’t work for them.

TORENCE: One of the biggest things is definitely trying to find ways to get things that they may need, whether it’s equipment or it could be a brace that they may need or a harness for whatever wheelchair.

NATALIE: It’s huge to be an advocate for your kid. It makes their life easier as they get older. Teaches them to advocate for themselves. Nobody knows your kid like you do and your kid knows that.

Close the transcript
Donovan, who was diagnosed with SMA Type 1, playing with his dad

Preparing for the future

Hear the perspectives of multiple families about why it’s important to be knowledgeable about SMA and its management even after treatment.

Maisie, who was diagnosed with SMA Type 1, saying, “Momma”

Maisie says, “Momma”

Watch as an SMA caregiver describes the moment her daughter said, “Momma” for the first time and why that single word meant so much for her to hear.

Making progress in SMA

Learn how newborn screening and having SMA treatments can help improve the lives of children with SMA.

Hear from SMA families

Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
  • 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?

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see any signs of a possible 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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

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 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 833-828-3947.

Please see the Full Prescribing Information.