Starting Treatment

Getting started on treatment.

Now that you and your doctor have decided on treatment with EXONDYS 51, you probably have questions about what comes next. We’ll guide you along the way so you’ll know just what to expect.





  1. Investigating Your Coverage

    Find out how SareptAssist, our patient support program, can help you get answers to questions about insurance benefits and verify coverage with your insurance company. Learn More

  1. Planning for Treatment

    Learn about creating a treatment plan that works for your family to help make the process go as smoothly as possible. Learn More

  1. About the Infusion

    Let’s take the mystery out of infusions. Here’s an overview of what they’re like, from start to finish. Learn More

  1. Ongoing Support

    It’s important to get ongoing support throughout treatment. SareptAssist will connect you with a dedicated Case Manager to help you start and stay on treatment. Learn More


Support, by their side.

In this video, Holly and her SareptAssist Case Manager, Ashley, talk about what it was like to get Holly’s son, Zyler, started on treatment with EXONDYS 51.

SareptAssist is a resource available only to those who have been prescribed EXONDYS 51 (eteplirsen). SareptAssist is only available in the U.S.



Ongoing support and information from Sarepta.






Sign Up

Icon Signup tips

Receive future tips and support about treatment with EXONDYS 51.

Icon Signup Informed

Stay informed on SareptAssist services.

An icon of 3 people and a circle

Find out about patient resources that are available.


Get updates about news and research on Duchenne from Sarepta.



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.

Read More


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).