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Vyondys 53

Generic name: golodirsen (GOE loe DIR sen)
Brand name: Vyondys 53
Drug class: Miscellaneous uncategorized agents

Medically reviewed by on Feb 18, 2020. Written by Cerner Multum.

What is Vyondys 53?

Vyondys 53 is used to treat Duchenne muscular dystrophy in adults and children who have a certain gene mutation. Your doctor will test you for this gene mutation.

Vyondys 53 was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some people responded to Vyondys 53, but further studies are needed.

Vyondys 53 may also be used for purposes not listed in this medication guide.


Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

Tell your doctor if you have ever had kidney problems.

Tell your doctor if you are pregnant or breastfeeding.

How is Vyondys 53 given?

Your doctor will perform blood tests to make sure you do not have conditions that would prevent you from safely using Vyondys 53.

Vyondys 53 is given as an infusion into a vein, usually once per week. A healthcare provider will give you this injection.

Vyondys 53 must be given slowly, and the infusion can take up to 60 minutes to complete.

Your kidney function may need to be checked while you are using this medicine.

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What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your Vyondys 53.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while receiving Vyondys 53?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Vyondys 53 side effects

Get emergency medical help if you have signs of an allergic reaction: hives, itching, rash, blistering or peeling; fever; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

  • pink, brown, or red urine;

  • foamy urine; or

  • swelling in your face, hands, feet, or stomach.

Common side effects may include:

  • headache;

  • fever;

  • stomach pain;

  • falls;

  • cough; or

  • runny or stuffy nose.

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 Vyondys 53?

Other drugs may affect Vyondys 53, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Frequently asked questions

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.