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Vyondys 53

Generic name: golodirsenGOE-loe-DIR-sen ]
Drug class: Miscellaneous uncategorized agents

Medically reviewed by on Feb 8, 2023. 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.

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.

Vyondys 53 may cause serious side effects. 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 of Vyondys 53 may include:

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.

Popular FAQ

Results from a small phase I/II clinical trial show that treatment with Vyondys 53 increases dystrophin protein levels within 48 weeks of starting treatment. Continue reading

Vyondys 53 is an antisense oligonucleotide that works by binding to exon 53 of dystrophin pre-mRNA, which results in this exon being skipped over during mRNA processing. Skipping of exon 53 allows cells to join different exons together to produce a dystrophin protein that is shorter but has some function. Continue reading

Vyondys 53 (golodirsen) is administered by intravenous (IV) infusion, which means it’s administered into a vein through a needle.

Vyondys 53 is given by a healthcare professional once a week. It is administered over 35 to 60 minutes via an in-line 0.2 micron filter. An indwelling venous access catheter is generally required. Continue reading

The new drugs approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) are Amondys 45 (casimersen), Viltepso (viltolarsen), Vyondys 53 (golodirsen), Exondys 51 (eteplirsen), and Emflaza (deflazacort).

Duchenne muscular dystrophy (DMD) is a genetic disease that causes progressive weakness, loss of motor function and damage to skeletal and heart muscles. DMD, which has no cure, primarily affects males starting in early childhood, usually between 2 and 3 years of age. Continue reading

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.