Resources and Events

For your information.

Learn. Connect. Get involved. If you’re ready to expand your knowledge of Duchenne muscular dystrophy or connect with the community face-to-face, you can get started here.

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Deletion of exons 48-50

Duchenne educational resources.

Getting Started on EXONDYS 51.  This guide can help you understand more about Duchenne, how exon skipping works, and how to get started on EXONDYS 51.

Download Brochure (PDF)


Duchenne community events.

Duchenne advocacy organizations often sponsor or participate in events that may be of interest to you. Find out more through the organizations listed below.

 

From Family & Caregiver Workshops to sporting events and fundraisers, Cure Duchenne provides ample opportunity to connect with others in the Duchenne community.

Learn more about Cure Duchenne events.

The JETT Foundation connects families, caregivers, and supporters of people living with Duchenne in New England for events throughout the year.

Learn more about JETT Foundation events.

The Muscular Dystrophy Association offers families and caregivers several ways to get involved, from Muscle Walks to fundraising events.

Learn more about MDA events.

Parent Project Muscular Dystrophy hosts events that provide opportunities for families, caregivers, and those living with Duchenne to gather, build support networks, and discuss all topics Duchenne.

Learn more about PPMD events.

 

 

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Related FAQs

Who can take EXONDYS 51?

Duchenne patients who receive EXONDYS 51 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 51. Your child's doctor is best equipped to determine if your child’s mutation is amenable to treatment with EXONDYS 51. We’ve developed a Doctor Discussion Guide to help you start that important conversation.

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.

See All FAQs

 

 

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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IMPORTANT RISK INFORMATION

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 www.fda.gov/medwatch 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).