Generic name: Golodirsen
Pregnancy & Lactation: Risk data not available
What is Vyondys 53?
Golodirsen 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.
Golodirsen was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some people responded to this medicine, but further studies are needed.
Golodirsen may also be used for purposes not listed in this medication guide.
Warnings
Tell your doctor if you have ever had kidney problems.
Tell your doctor if you are pregnant or breastfeeding.
How should I use Vyondys 53
Use Vyondys 53 (Intravenous) exactly as directed on the label, or as prescribed by your doctor. Do not use in larger or smaller amounts or for longer than recommended.
Your doctor will perform blood tests to make sure you do not have conditions that would prevent you from safely using golodirsen.
Golodirsen is given as an infusion into a vein, usually once per week. A healthcare provider will give you this injection.
This medicine 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.
Call your doctor for instructions if you miss an appointment for your golodirsen injection.
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?
Follow your doctor's instructions about any restrictions on food, beverages, or activity.