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Home Knowledge Center Wellness Library onasemnogene abeparvovec (Zolgensma)

onasemnogene abeparvovec (Zolgensma)

Pronunciation: ON a SEM noe jeen A be PAR voe vek

What is the most important information I should know about onasemnogene abeparvovec?

What is the most important information I should know about onasemnogene abeparvovec?

This medicine can cause serious liver damage, especially in children who already have liver problems. Your doctor will perform blood tests to make sure your child does not have conditions that would make it unsafe to receive onasemnogene abeparvovec.

What is onasemnogene abeparvovec?

What is onasemnogene abeparvovec?

Onasemnogene abeparvovec is used to treat spinal muscular atrophy in children younger than 2 years old who have a specific gene mutation that affects nerve signals to the muscles.

This medicine may not be effective in a child who is completely paralyzed or is dependent on a ventilator for breathing.

Onasemnogene abeparvovec may also be used for purposes not listed in this medication guide.

What should I discuss with my healthcare provider before receiving onasemnogene abeparvovec?

What should I discuss with my healthcare provider before receiving onasemnogene abeparvovec?

This medicine can cause serious liver damage. Tell your doctor if your child has ever had liver problems.

It may not be safe to breastfeed your child while he or she is receiving this medicine. Ask your doctor about any risk.

How is onasemnogene abeparvovec given?

How is onasemnogene abeparvovec given?

Your doctor will perform blood tests to make sure your child does not have conditions that would make it unsafe to receive onasemnogene abeparvovec.

Onasemnogene abeparvovec is given as an infusion into a vein, usually only one time. A healthcare provider will give your child this injection.

This medicine must be given slowly, and the infusion can take about 1 hour to complete.

Your child may be given steroid medication to help prevent serious side effects of onasemnogene abeparvovec. Keep giving your child the steroid for as long as your doctor has prescribed. The doctor may gradually reduce the dose as your child continues taking the steroid.

Your child will need frequent blood tests to check his or her liver function.

Your child may need to be vaccinated against respiratory syncytial (sin-SISH-ul) virus, or RSV. Follow the doctor's instructions about this vaccination.

If your child vomits shortly after taking the steroid medicine, do not take another dose. Call the doctor for instructions.

What happens if I miss a dose?

What happens if I miss a dose?

Onasemnogene abeparvovec is used as a single dose and does not have a daily dosing schedule.

What happens if I overdose?

What happens if I overdose?

Since this medicine is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while receiving onasemnogene abeparvovec?

What should I avoid while receiving onasemnogene abeparvovec?

If your child is also taking steroid medicine, the child should not receive certain vaccines. If your child is due for any vaccine, ask your doctor first before your child gets the shot(s).

Onasemnogene abeparvovec can pass into your child's stools (bowel movements). For at least 1 month after your child receives a dose, avoid allowing the child's stools to come into contact with hands or other surfaces. Wear rubber gloves while cleaning up the child's body fluids, handling contaminated trash or laundry, or changing diapers. Seal used diapers in disposable bags and throw them into the trash. Wash hands before and after removing gloves. Wash soiled clothing and linens separately from other laundry.

What are the possible side effects of onasemnogene abeparvovec?

What are the possible side effects of onasemnogene abeparvovec?

Get emergency medical help if your child has signs of an allergic reaction: hives; difficult breathing; swelling of the face, lips, tongue, or throat.

Call your doctor at once if your child has:

  • jaundice (yellowing of the skin or eyes); or
  • easy bruising, unusual bleeding, purple or red spots under your skin.

Common side effects may include:

  • vomiting; or
  • abnormal liver function tests.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What other drugs will affect onasemnogene abeparvovec?

What other drugs will affect onasemnogene abeparvovec?

Other drugs may affect onasemnogene abeparvovec, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your child's current medicines and any medicine the child starts or stops using.

Where can I get more information?

Where can I get more information?

Your doctor or pharmacist can provide more information about onasemnogene abeparvovec.

This information does not replace the advice of a doctor. Healthwise, Incorporated, disclaims any warranty or liability for your use of this information. Your use of this information means that you agree to the Terms of Use. Learn how we develop our content.

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