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During your EXONDYS 51 treatment journey, it’s important to stay informed. We’re committed to keeping you updated with information on treatment, education, and support.

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Meet Liam, age 19
Deletion of exons 48-50


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SareptAssist is designed to provide the information you need to navigate the process of starting and staying on therapy. Your Case Manager will work closely with you to provide one-on-one, personalized support.

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The Duchenne muscular dystrophy community is informative, understanding, and well connected. And they are ready to help with the kind of support that comes only from people who know what you’re going through.

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Related FAQs

Will my insurance cover EXONDYS 51?

Once your child is prescribed EXONDYS 51, you may enroll in SareptAssist, our patient support program. Your Case Manager will start a benefits investigation on your current insurance plan and can help explain details about your coverage. How SareptAssist can help.

What support is available for people being treated with EXONDYS 51?

We developed SareptAssist, our patient support program, to help patients start and stay on therapy. You’ll have support at every step—from managing insurance approvals to coordinating drug delivery. How SareptAssist can help.



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WHAT IS EXONDYS 51 (eteplirsen)?

EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51. EXONDYS 51 was approved under accelerated approval. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.

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Allergic reactions, including wheezing, chest pain, cough, rapid heart rate, and hives have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

The most common side effects seen in greater than 10% of patients receiving EXONDYS 51 (N=163) in other clinical trials were headache, cough, rash, and vomiting. 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

Please see the full Prescribing Information for EXONDYS 51 (eteplirsen).