The Duchenne Community

Finding strength in community.


When your child is newly diagnosed with Duchenne muscular dystrophy, you may not know for sure what’s ahead, but many others in the community do. Connect with patients and parents of children with Duchenne for the kind of support you can’t get anywhere else.

Community

Start connecting.

The groups below—many founded by parents of children with Duchenne—help connect patients and families to education, research, support services, and the broader Duchenne community. Here are a just a few of the organizations that work to raise both awareness of Duchenne and funds directed toward research while providing programs and information to help individuals with Duchenne. 

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Get help with genetic testing.

If your child has been diagnosed with Duchenne and you’ve been unable to access genetic testing because of financial barriers, there’s a program that may be able to help. Decode Duchenne offers free genetic testing to the Duchenne community and can provide genetic counselors to interpret test results.

Decode Duchenne is administered by Parent Project Muscular Dystrophy and The Duchenne Registry. Decode Duchenne is sponsored by Sarepta Therapeutics and PTC Therapeutics.

Visit Decode Duchenne to learn more.

 

 

 

“My advice for other parents is to reach out, ask questions, and get support.”

–Holly, Zyler’s mom

Related FAQs

Why is genetic testing important?

A genetic test will confirm a diagnosis of Duchenne and allow your child’s doctor to identify the genetic mutation so treatment options can be considered and to assist with family planning. Understanding genetic testing.

We have a genetic test. How do I know if my child can be treated with EXONDYS 51?

A doctor will need to interpret the test results to consider appropriate treatment options. Learn more about deletions.

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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Ongoing support and information from Sarepta.

 

 

 

 

 

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Receive future tips and support about treatment with EXONDYS 51.

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Stay informed on SareptAssist services.

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Find out about patient resources that are available.

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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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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).