Dosage Details for Zolgensma

This section describes the usual dosage of Zolgensma. Keep reading to learn more.

What is Zolgensma’s form?

Zolgensma is given as an intravenous (IV) infusion (an injection into your vein over a period of time). Your child will receive their infusion at a doctor’s office, clinic, or hospital.

What strength does Zolgensma come in?

Zolgensma is available in one strength: 2 x 10¹³ vector genomes (vg)* per milliliter (mL) of suspension.

What are the usual dosages of Zolgensma?

The information below describes the Zolgensma dosage that’s commonly used or recommended.

What’s the dosage of Zolgensma for children with spinal muscular atrophy?

Zolgensma is used to help treat spinal muscular atrophy (SMA) in children younger than 2 years old.

The recommended dosage for children is 1.1 x 10¹⁴ vg per kilogram (kg)* of body weight.

Zolgensma is given as a one-time infusion over a period of 60 minutes.

Starting the day before they receive Zolgensma, your child will begin taking an oral liquid form of a corticosteroid. This helps reduce inflammation, which lowers the risk of liver damage. They’ll continue taking the corticosteroid for at least 30 days.

Your child’s doctor will continue to monitor their liver function after they receive Zolgensma. If they don’t have symptoms of liver problems, their doctor will slowly lower the corticosteroid dosage over a period of 28 days until they can stop taking it.

Your child’s liver function will be monitored for at least 3 months after they receive Zolgensma.

For more information about Zolgensma’s dosage, talk with your child’s doctor.

* One kilogram (kg) is about 2.2 pounds (lb).

Is Zolgensma used long term?

No. Zolgensma is used as a one-time treatment for SMA. But your child will likely receive an oral corticosteroid medication starting one day before they receive Zolgensma and for at least 30 days. Your child’s doctor will monitor their liver function for at least 3 months after Zolgensma is given.

Zolgensma is given as an IV infusion over a period of 60 minutes.

Your child will receive the infusion at a doctor’s office, clinic, or hospital. To help reduce inflammation, your child will receive an oral corticosteroid medication starting 1 day before they receive Zolgensma and for at least 30 days in total.

If you have questions about your child’s dosage or how they’ll receive Zolgensma, talk with their doctor.

Below are answers to some commonly asked questions about Zolgensma’s dosage.

Is Zolgensma’s dosage similar to the dosages of Evrysdi or Spinraza?

No. The forms and how often they’re given are different.

Zolgensma, risdiplam (Evrysdi), and nusinersen (Spinraza) are all used to treat spinal muscular atrophy (SMA) in certain children. But Evrysdi and Spinraza are also used in adults, while Zolgensma is not.

Evrysdi comes as an oral liquid solution. Spinraza is given as an injection by a healthcare professional. Both of these drugs may be used long term to treat SMA. Zolgensma, on the other hand, is given as a one-time IV infusion by a healthcare professional.

Your child’s doctor will prescribe the drug and dosage that’s right for them. To learn more about how these drugs compare, talk with your child’s doctor.

How long does it take for Zolgensma to start working?

Zolgensma starts to work after your child receives their dose, but it may take a while before their symptoms begin to ease. In studies of Zolgensma, some children had their SMA symptoms ease within 1 month after receiving the medication.

If you have other questions about what to expect from Zolgensma treatment, talk with your child’s doctor.

The sections above describe the usual dosage provided by Zolgensma’s manufacturer. If your doctor recommends this drug for your child, they’ll prescribe the dosage that’s right for them.

Talk with your child’s doctor if you have questions or concerns about their Zolgensma dosage.

Examples of questions you may want to ask include:

• Will my child receive their Zolgensma dosage with other drugs used for SMA?
• Can my child receive a second dose of Zolgensma if the first dose doesn’t improve their symptoms?
• Will my child need a dosage adjustment for Zolgensma if they have kidney problems?

To learn more about Zolgensma, see these articles:

• Zolgensma (onasemnogene abeparvovec-xioi)
• Zolgensma and Cost: What You Need to Know

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Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.