Getting Support

We’re in this together.

When you receive a diagnosis of a rare disease like Duchenne muscular dystrophy, you may feel quite alone in the world. At Sarepta, we want you to know about the wide range of support that’s available to you—from SareptAssist, our comprehensive patient support program, to national patient advocacy groups that provide education and resources for your entire family.


Meet Liam, age 19
Deletion of exons 48-50

SareptAssist: Support, by your side.

We recognize that families of children receiving treatment for Duchenne muscular dystrophy need more than just medicine. When your child is prescribed EXONDYS 51, we’ll connect you with a dedicated SareptAssist Case Manager who helps with the treatment process. You’ll have support at every step—from managing insurance approvals to coordinating drug delivery. 

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Connect with the Duchenne community.

Duchenne muscular dystrophy is a rare disease—but you’re not facing it alone. Many families have been in your shoes, and over the years, they’ve created a strong community of patient advocates, educators, and service providers. 

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Resources on Duchenne.

Find educational resources that may be of interest to you.

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Ongoing support and information from Sarepta.






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Receive future tips and support about treatment with EXONDYS 51.


Stay informed on SareptAssist services.


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.

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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).