Vyondys 53 (golodirsen) is a brand-name drug that’s prescribed for Duchenne muscular dystrophy (DMD) in adults and children with a certain gene mutation. Vyondys 53 comes as an intravenous infusion that’s given by a healthcare professional once weekly.

Vyondys 53 is approved by the Food and Drug Administration (FDA)* to treat Duchenne muscular dystrophy (DMD) in adults and children who have a certain gene mutation. DMD is a genetic disorder that most often affects children and young adults and causes muscle loss and weakness over time.

Vyondys 53 belongs to a drug class called antisense oligonucleotides. Vyondys 53 is not available in a generic version.

Keep reading for specific information about the dosage of Vyondys 53, including its strength and how to receive the medication. For a comprehensive look at Vyondys 53, see this article.

Note: This article describes typical dosages for Vyondys 53 provided by the drug’s manufacturer. However, your doctor will prescribe the Vyondys 53 dosage that’s right for you.

* For this use, Vyondys 53 received accelerated approval from the FDA. Accelerated approval is based on the results of early clinical trials of the drug. The FDA will decide on full approval of Vyondys 53 for this use after more trials have been completed.

Read below for details about Vyondys 53’s dosage for its approved uses.

Vyondys 53 form

Vyondys 53 comes as a solution in a single-dose vial. The solution is mixed with a liquid to form a diluted solution for intravenous (IV) infusion. It’s always given by a healthcare professional.

Vyondys 53 strength

Vyondys 53 comes in one strength of 50 milligrams per milliliter (mg/mL).

Typical dosages

The following information describes dosages that are commonly prescribed or recommended. Your doctor will determine the best dosage to fit your needs.

Dosage for Duchenne muscular dystrophy

Doctors may prescribe Vyondys 53 to treat Duchenne muscular dystrophy (DMD).

If your doctor prescribes Vyondys 53 for DMD, your dose will be based on your body weight. The recommended dosage of Vyondys 53 is 30 milligrams of drug per kilogram of body weight (mg/kg). One kg equals about 2.2 pounds (lb). It’s given by a healthcare professional once weekly as a 35- to 60-minute IV infusion.

For more information about your specific dosage, talk with your doctor.

Children’s dosage

Vyondys 53 is approved to treat DMD in children.

Like adults, the dosage is based on the child’s body weight in kilograms (kg). Your child will receive 30 mg/kg of Vyondys 53. It’s given by a healthcare professional as an intravenous infusion over 35–60 minutes once weekly.

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

Long-term treatment

Vyondys 53 is meant to be given as a long-term treatment. If you and your doctor determine that Vyondys 53 is safe and effective for you, you’ll likely receive it long term.

Before you start receiving Vyondys 53, your doctor will discuss your treatment plan with you.

Your doctor or another healthcare professional will give you the intravenous (IV) infusion of Vyondys 53. First, they’ll determine your dosage based on your body weight in kilograms. Then, they’ll dilute the Vyondys 53 solution with another liquid and prepare the infusion. You’ll likely go to your doctor’s office, an infusion center, or a hospital to receive your infusion. The infusion takes about 35–60 minutes.

If you have other questions about how Vyondys 53 is given, talk with your doctor or pharmacist. There’s also more information about what to expect with your infusion on the manufacturer’s website.

If you miss your appointment for a Vyondys 53 infusion, call your doctor’s office as soon as possible to reschedule. They’ll adjust your dosing schedule as needed.

If you need help remembering your appointments, try setting an alarm or downloading a reminder app on your phone.

Below are some frequently asked questions about Vyondys 53.

Is the dosage of Vyondys 53 similar to the dosage of Exondys 51?

Yes, the forms, dosage, and how often you receive each drug are similar. Vyondys 53 and Exondys 51 (eteplirsen) are both intravenous infusions given once weekly for Duchenne muscular dystrophy (DMD). The dosage for both these drugs is 30 mg/kg of body weight.

To learn more about how these drugs compare and which one is right for you, talk with your doctor.

How long does it take for Vyondys 53 to start working?

Vyondys 53 starts to work after your first dose. Because of how the drug works, you likely won’t feel the drug working in your body. But your doctor will monitor you during treatment to check whether the drug is working to treat your condition.

Talk with your doctor if you have questions about what to expect with Vyondys 53 treatment.

The dosages in this article are typical dosages provided by the drug’s manufacturer. If your doctor recommends Vyondys 53 for you, they will prescribe the dosage that’s right for you. Always follow the dosage that your doctor prescribes.

As with any drug, never change your dosage of Vyondys 53 without your doctor’s recommendation. If you have questions about the dosage of Vyondys 53 that’s best for you, talk with your doctor.

Besides learning about dosage, you may want other information about Vyondys 53. These additional articles might be helpful:

  • More about Vyondys 53. For information about other aspects of Vyondys 53, refer to this article.

Disclaimer: Medical News Today 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.