Vyondys 53 (golodirsen) is a prescription drug used to treat Duchenne muscular dystrophy. The drug comes as a liquid solution that’s typically given once per week by intravenous (IV) infusion.
Vyondys 53 is used to treat Duchenne muscular dystrophy in adults and children with a certain genetic mutation. Your doctor will order a genetic test to determine whether this medication is right for you.
The active ingredient in Vyondys 53 is golodirsen. (An active ingredient is what makes a drug work.) Vyondys 53 belongs to a group of drugs called antisense oligonucleotides.
This article describes the dosage of Vyondys 53, as well as its strength and how it’s given. To learn more about Vyondys 53, see this in-depth article.
The table below highlights the basics of Vyondys 53’s dosage.
|liquid solution given by intravenous (IV) infusion (an injection into a vein given over time)
|50 milligrams (mg) per milliliter (mL) of solution
|30 mg per kilogram (kg) of body weight, once weekly
Keep reading for more details about Vyondys 53’s dosage.
What is Vyondys 53’s form?
Vyondys 53 comes as a liquid solution given by IV infusion.
What strength does Vyondys 53 come in?
Vyondys 53 comes in one strength of 50 mg/mL of solution.
What are the usual dosages of Vyondys 53?
The information below describes the dosage most commonly used or recommended. But your doctor will determine the best dosage to fit your needs.
Dosage for Duchenne muscular dystrophy (DMD)
The typical Vyondys 53 dosage for adults with DMD is 30 mg per kg of body weight, administered once weekly.
What’s the dosage of Vyondys 53 for children?
Vyondys 53 is used to treat DMD in children. The dosage for children is the same as the dosage for adults. This is 30 mg per kg of body weight once per week.
For more information about the dosage of Vyondys 53 for children, talk with your child’s doctor or a pharmacist.
Is Vyondys 53 used long term?
Yes, Vyondys 53 is usually a long-term treatment. If you and your doctor determine that it’s safe and effective for your condition, you’ll likely receive it long term.
The dosage of Vyondys 53 you’re prescribed may depend on various factors. These include:
- your body weight
- side effects you may have with Vyondys 53 treatment
Vyondys 53 comes as a liquid solution that’s given as an IV infusion. You’ll usually receive infusions at your doctor’s office or a clinic. In some cases, it may also be possible for a healthcare professional to give you infusions at home.
The infusion will usually be given into a vein in your arm. The healthcare professional may numb the area with a local anesthetic cream before inserting the needle into your vein.
Each infusion lasts about 35–60 minutes. But if you have an allergic reaction to the infusion, the healthcare professional may slow down or pause the infusion while they treat your symptoms. If this happens, your next infusion may be given over a longer period of time.
To learn more about allergic reactions to Vyondys 53, see this article.
If you miss an appointment to receive a dose of Vyondys 53, call your doctor’s office as soon as possible to reschedule. If you need help remembering your appointments, try downloading a reminder app on your phone.
Below are answers to some commonly asked questions about Vyondys 53’s dosage.
Is Vyondys 53’s dosage similar to that of Exondys 51?
Yes, it’s similar. Vyondys 53 and Exondys 51 both come as a liquid solution given by IV infusion once per week. Each drug is given by a healthcare professional at a doctor’s office or clinic.
The dose in milligrams for each drug is also similar. Your doctor will prescribe the drug and the dosage that’s right for you.
To learn more about how these drugs compare, see this article or talk with your doctor.
How long does it take for Vyondys 53 to start working?
Vyondys 53 starts to work in your body after your first dose. But it’s not known how long it may take for your symptoms to ease. Because of how the drug works, you likely won’t feel it working in your body. But your doctor will monitor you during treatment to check whether the drug is effectively treating your condition.
If you have other questions about what to expect from your Vyondys 53 treatment, talk with your doctor.
The sections above describe the usual dosage provided by the manufacturer of Vyondys 53. If your doctor recommends this drug, they’ll prescribe the dosage that’s right for you. Talk with them if you have questions or concerns about your current dosage. Here are some examples of questions you may want to ask:
- Would a higher dosage of Vyondys 53 be more effective for my condition?
- Will my dosage of this drug change if I have side effects?
- How can I prepare my child for receiving Vyondys 53 infusions?
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Disclaimer: Healthline 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.