What is Exondys 51?
Exondys 51 helps the body produce a dystrophin protein. Dystrophin, a protein that helps stabilize muscle tissue structure and is essential for muscle development and function, is needed. Muscle fibers become weaker and more easily damaged without dystrophin. Walking, for example, can be affected by this.Children with Duchenne muscle dystrophy are affected by a mutation in a gene that would normally trigger the body to make dystrophin.Exondys is prescribed to children who have Duchenne muscle dystrophy. Exondys is not a treatment for DMD. However, it may help to reduce muscle weakness and muscle loss caused by the disease.Exondys 51 has been approved "accelerated" by the US Food and Drug Administration. Some children responded positively to this medication in clinical studies. Further studies are required to determine whether this medicine is effective for treating DMD.Exon 50 can be used in other ways not mentioned in this guide.
Side effects of Exondys 51
If your child shows any of the following symptoms of an allergic reaction, seek immediate medical attention: hives, difficulty breathing, swelling of the face, lips, or throat.
Exondy 50 may cause the following side effects:
- Pain, bruising, or itching; skin rash; or any other irritation at the injection site
- Vomiting;
- Joint pain
- Problems with balance or
- Cold symptoms include stuffy nose and sneezing.
There may be other side effects. For medical advice on side effects, call your doctor.Contact the FDA by dialing 1-800-FDA-1088 in order to report adverse side effects.
Similar/related drugs
Viltepso, Amondys 45, Emflaza, Vyondys, Elevidys, Deflazacort, and Delandistrogene Moxeparvovec
Warnings
Tell your doctor all of your child's medical problems or allergies and any medicines they use before your child is prescribed Exondys.
Before you take this drug
Inform your doctor if your child has had a severe reaction to any previous Exondys51 injections.Tell your doctor all of your child's medical problems or allergies and any medicines they use before you give Exondys to your child.Exondys 51 is a drug that has been used mostly in children. However, it's not known if Exondys 51 can be harmful to a woman who is pregnant or nursing.
How to take Exondys 51?
Exondys is administered as an intravenous infusion. This injection will be given by a healthcare provider.A numbing agent can be applied to the area of skin where the IV needle is going to be placed.Exondys is administered once a week. The infusion of this medicine can take as long as an hour.When Exondys51 is injected, tell your caregiver if you experience chest tightness, itchiness, warmth, or lightheadedness.Inform your doctor of any weight changes. Exondys doses are calculated based on your child's weight. Any changes in weight may change the dose.
What happens if I miss the dose?
If you missed an Exondys51 appointment, call your doctor to get instructions.
What happens if I overdose?
Call 1-800-222-1222 for poison help or seek immediate medical attention.
What should be avoided?
If your doctor prescribes any restrictions regarding food, beverage, or activity, follow their instructions.
Interaction with other drug
Exondys 50 may be affected by other drugs, such as prescription and over-the-counter medicines, vitamins, and herbal products. Inform your child's health care providers of all the medicines he or she is taking, as well as any new medications.
