Zolgensma Dosage: Form, Strength, How it’s Given, and More
Zolgensma (onasemnogene abeparvovec-xioi) is a prescription gene therapy drug that’s used to treat spinal muscular atrophy in certain children. A healthcare professional will administer the drug as a one-time intravenous (IV) infusion.
Boxed warning: Risk of liver damage
Zolgensma has a boxed warning about the risk of liver damage. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). It alerts doctors and patients about drug effects that may be dangerous.
Zolgensma can cause sudden liver failure and death. It can also cause liver injury و elevated liver enzymes. If your child already has liver problems, they may have a higher risk of liver damage with Zolgensma.
Your child’s doctor will check their liver function before administering Zolgensma and for at least 3 months after the medication is given.
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 take the corticosteroid for at least 30 days. If your child shows any signs of liver problems, they’ll continue taking corticosteroids until their liver function improves. Their dosage of the corticosteroid will likely be reduced over a period of 28 days before they stop taking it.
What is Zolgensma’s dosage?
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.
How is Zolgensma administered?
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.
Frequently asked questions
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.
What should I ask my 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:
To learn more about Zolgensma, see these articles:
Great post! Zolgensma’s dosage details are crucial for understanding its administration. The clear breakdown of form, strength, and delivery method really helps clarify how this gene therapy works. Thanks for making this complex information accessible!
Thanks for the detailed breakdown on Zolgensma dosage! It’s really helpful to understand the form, strength, and administration process for this treatment. This information is crucial for anyone considering or already undergoing this therapy. Appreciate the clarity and depth you provided!
Great article! It’s incredibly informative and breaks down complex information about Zolgensma dosage into easy-to-understand segments. I appreciate the detailed explanations on the form, strength, and administration method. This will be really helpful for parents and caregivers of children with SMA. Thanks for sharing!
Absolutely! It’s fascinating to see advancements like Zolgensma in the field of gene therapy. Knowing how it’s administered and the specifics of its dosage and strength can be particularly helpful for those needing this groundbreaking treatment. Thanks for providing such detailed and valuable information!
Thank you for this comprehensive breakdown on Zolgensma dosage! It’s incredibly helpful to understand the form, strength, and administration process clearly. For anyone facing decisions about treatment, this kind of detailed information is invaluable. Keep up the great work in educating and supporting your readers! 🙏