Exondys 51 (eteplirsen) is an injectable medication that's used for a rare, genetic condition called Duchenne Muscular Dystrophy (DMD). It was the first medication approved for DMD through the FDA's Accelerated Approval Program. Exondys 51 (eteplirsen) is given as an intravenous (IV) infusion by a healthcare provider. Your child will need to take a genetic test before starting this medication to make sure it's right for them.
Treatment of Duchenne Muscular Dystrophy (DMD) in people with a specific genetic mutation
Exondys 51 (eteplirsen) is an antisense oligonucleotide. It attaches to exon 51, a part of the gene that makes dystrophin. Dystrophin is a protein that keeps muscle cells intact and strong. In people with DMD, this protein is missing because of a mutation in exon 51. Exondys 51 (eteplirsen) works by exon skipping, meaning it cuts out exon 51 and its mutation from the finished protein. This helps to make a shorter but more functional dystrophin, which potentially provides strength to muscles and relief from symptoms of DMD.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
Can help some people with DMD who never had a treatment option before
Only needs to be injected once a week so it's convenient for parents with a busy schedule
Doesn't interact with other medications or supplements
Only available as a brand medication, so it might be expensive
More likely to cause severe allergic reactions
Requires IV access, so it's not as convenient as taking a pill
It takes about 35 minutes to 1 hour to give the full dose of Exondys 51 (eteplirsen). Most commonly, your child will receive this medication at an infusion center or a provider's office. In some cases, it can be given at home by a home health nurse.
It's best to get your Exondys 51 (eteplirsen) injection around the same day every week. If you miss an infusion, talk with your provider right away about rescheduling your appointment.
If you have pain around the injection site after receiving Exondys 51 (eteplirsen), let your provider know. They might suggest using a topical cream on the area before injection to ease the pain and discomfort from the needle.
Tell your provider if your child experiences any changes in weight while receiving Exondys 51 (eteplirsen) because the dose might need to be changed.
Exondys 51 (eteplirsen) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Exondys 51 (eteplirsen) can cause allergic reactions, which can be life-threatening. Watch out for signs and symptoms of allergic reactions, such as trouble breathing, wheezing, chest pain, cough, fast heartbeat, and hives. Let your provider know right away if allergic reactions occur. Your provider might slow the infusion or stop the treatment temporarily to help with your symptoms.
Exondys 51 (eteplirsen) is an intravenous (IV) infusion that is administered by a healthcare provider. The dose depends on your weight and your provider will determine the appropriate dose for you each time.
In general, the typical dose is 30 mg/kg once a week injected into a vein.
By providing your email, you consent to receive marketing communications from GoodRx, which may include content and/or data related to men’s health, women's health, reproductive care, or sexual health. You agree to the GoodRx Terms of Use and acknowledge the Privacy Policy. You can unsubscribe at any time.
Research prescriptions and over-the-counter medications from A to Z, compare drug prices, and start saving.