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 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 and Detect Muscular Dystrophy offer 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 or Detect Muscular Dystrophy to learn more.

 


 

Decode Duchenne

 

Detect DMD

 

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 VYONDYS 53?

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 VYONDYS 53?

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 VYONDYS 53.

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Stay informed about 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 VYONDYS 53 (golodirsen)?

VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

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IMPORTANT RISK INFORMATION

Allergic reactions, including rash, fever, itching, hives, and inflammation and/or peeling of the skin have occurred in patients who were treated with VYONDYS 53. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Damage to the kidneys was seen in animals who received golodirsen. Although damage to the kidneys was not seen in clinical studies with VYONDYS 53, potentially fatal kidney damage has occurred with other drugs that work in a similar way. Your doctor may recommend urine and blood testing before starting treatment followed by urine testing every month and a blood test every 3 months to monitor your kidneys.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, fever, itching, hives, and inflammation and/or peeling of the skin have occurred in patients who were treated with VYONDYS 53.  Seek immediate medical care if signs and symptoms of allergic reactions occur.

Damage to the kidneys was seen in animals who received golodirsen. Although damage to the kidneys was not seen in clinical studies with VYONDYS 53, potentially fatal kidney damage has occurred with other drugs that work in a similar way. Your doctor may recommend urine and blood testing before starting treatment followed by urine testing every month and a blood test every 3 months to monitor your kidneys.

Adverse reactions that have occurred in at least 20% of patients treated with VYONDYS 53 and more often than in patients who received an inactive intravenous (IV) infusion were headache (41%, 10%), fever (41%, 14%), fall (29%, 19%), pain in the abdomen (27%, 10%), infection of the nose and throat (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).

Other adverse reactions that occurred in greater than 5% of patients treated with VYONDYS 53 and more often than in patients who received an inactive IV infusion were pain at the IV site, back pain, pain, diarrhea, dizziness, stretch or tear in a ligament, bruising, flu, pain in the mouth and throat, stuffy or runny nose, scrapes or scratches of the skin, ear infection, seasonal allergy, fast heartbeat, reactions related to the IV catheter site, constipation, and broken bones.

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 VYONDYS 53 (golodirsen).