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.

Nicholas

Meet Nicholas, age 17
Amenable to exon 53 skipping

Duchenne educational resources.


Getting Started on VYONDYS 53.  This guide can help you understand more about Duchenne, how exon skipping works, and how to get started on VYONDYS 53.

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 and 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.

Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.

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 VYONDYS 53?

Duchenne patients who receive VYONDYS 53 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 53. Your child's doctor is best equipped to determine if your child’s mutation is amenable to treatment with VYONDYS 53. We’ve developed a Doctor Discussion Guide to help you start that important conversation.

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.

See All FAQs

 

 

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).