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Overview: Vyondys 53 is used to treat Duchenne muscular dystrophy (DMD) in people who have a specific mutation in the DMD gene. This medicine helps these people make a shorter but working protein called dystrophin that is needed for muscles to work. Common side effects include headache, fever, stomach pain, and nausea. Tell your health care provider right away if you have confusion, unusual swelling, or a reduced need to pee. Vyondys 53 is injected into your vein (IV) by your health care provider.

Uses

What is Vyondys 53 used for?

Vyondys 53 (golodirsen) is commonly used to treat Duchenne muscular dystrophy (DMD).

Vyondys 53 may also be used for other conditions as determined by your health care provider.

How does Vyondys 53 work (mechanism of action)?

Dystrophin is a protein that is needed for muscles to work. Some people with Duchenne muscular dystrophy have a specific change in the DMD gene that can be treated with a process called exon 53 skipping. Vyondys 53 is a medicine that helps these people make a shorter but working dystrophin protein.

How is Vyondys 53 supplied (dosage forms)?

Brands and other names

  • Vyondys 53

Dosage forms and strengths

Vyondys 53 is available in an injectable form that is given by a health care provider.

How should I store Vyondys 53?

Vyondys 53 is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Vyondys 53?

The most common side effects of Vyondys 53 are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Headache
  • Fever
  • Falls
  • Stomach pain
  • Infection in the nose or throat, sore throat, cough, or common cold symptoms
  • Nausea or vomiting

There may be other side effects of Vyondys 53 that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088. In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Vyondys 53?

While less common, the most serious side effects of Vyondys 53 are described below, along with what to do if they happen.

Severe Allergic Reactions. Vyondys 53 may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be pale, red, or skin colored depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Kidney Damage. Kidney damage may happen when using  Vyondys 53. Call your health care provider right away if you have any of the following symptoms of kidney damage.

  • Reduced need to pee
  • Swelling in your feet, ankles, or legs
  • Weakness or unusual tiredness
  • Difficulty catching your breath or chest pain/pressure
  • Confusion
  • Nausea
  • Seizures

Warnings & Precautions

Who should not use Vyondys 53?

Allergies to Ingredients. People who are allergic to any of the following should not use Vyondys 53.

  • Vyondys 53
  • Golodirsen
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Vyondys 53.

What should I know about Vyondys 53 before using it?

Vyondys 53 will be injected into your vein (intravenous) by a health care provider in a hospital or clinic.

Keep all your appointments with your health care provider, including lab appointments. They will use these appointments to check how well your kidneys are working and other health care markers while using Vyondys 53.

What should I tell my health care provider before using Vyondys 53?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them determine if Vyondys 53 is right for you.

In particular, make sure that you discuss any of the following.

Kidney problems. Tell your health care provider if you have kidney problems.

Pregnancy. It is not known if or how Vyondys 53 could affect a pregnancy or harm a fetus. Tell your health care provider if you are pregnant or plan to become pregnant.

Breastfeeding. It is not known if Vyondys 53 passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed.

How can I get more information about Vyondys 53?

For more information about Vyondys 53, you can visit the drugmaker’s website at www.vyondys53.com or call them at 888-727-3782.

Interactions

Does Vyondys 53 interact with foods or drinks?

There are no known interactions between Vyondys 53 and foods or drinks.

It is unknown if drinking alcohol will affect Vyondys 53.

Does Vyondys 53 interact with other medicines (drug interactions)?

There are no known interactions between Vyondys 53 and other medicines. Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Vyondys 53?

If you or someone else has used too much Vyondys 53, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Vyondys 53?

If you miss an appointment to get your Vyondys 53 dose, call to make another appointment as soon as possible.