About Duchenne

Duchenne muscular dystrophy: the most common muscular dystrophy in children.

What does it mean when a child is diagnosed with Duchenne muscular dystrophy (DMD)? Arming yourself with information about the cause and progression of this rare disease may help you make important decisions moving forward.

Meet Luke, age 12
Amenable to exon 53 skipping

Understanding the cause and symptoms of Duchenne.

Duchenne muscular dystrophy is often referred to as DMD, or just Duchenne, and affects mostly boys. It is a progressive disease that causes muscle weakness that affects many parts of the body. Learn the common symptoms and the underlying cause behind this rare condition and why informed decision making at the earliest stages is so critical.

Learn More About Duchenne

Genes, dystrophin, and Duchenne.

Why are some children born with Duchenne? Putting it simply, Duchenne is caused by a genetic mutation that limits their body’s production of a protein called dystrophin. There is much more to learn about genetic mutations, including which specific mutations cause Duchenne, the role of dystrophin in muscle function, and why it’s so important to know your child’s specific mutation.

See How Genes Play a Role

Andres, age 9 Amenable to exon 53 skipping

The path to a Duchenne diagnosis.

Duchenne is initially diagnosed through a creatine kinase (CK) blood test. But too often, children who display symptoms of Duchenne are not given this simple test, delaying their diagnosis for years. While the CK test is a highly accurate indicator of Duchenne, genetic testing is required to confirm a diagnosis. The good news is that today’s genetic tests reveal far more information—including the exact mutation in the dystrophin gene—than those performed just a few years ago.

Get Details on Diagnosis

Ongoing support and information from Sarepta.

Receive future tips and support about treatment with VYONDYS 53.

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Find out about patient resources that are available.

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.

IMPORTANT SAFETY INFORMATION

Contraindications: Do not receive VYONDYS 53 if you are allergic to golodirsen or any of the ingredients in VYONDYS 53. Serious allergic reactions to golodirsen have included anaphylaxis, which may include difficulty breathing and tightness in the chest.

Hypersensitivity Reactions: Serious allergic reactions, including anaphylaxis, have occurred in patients who were treated with VYONDYS 53. Additional 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. Patients should seek immediate medical care should they experience signs and symptoms of allergic reaction. Your doctor will institute appropriate medical treatment which may include slowing, interrupting, or discontinuing the VYONDYS 53 infusion. Your doctor will monitor you until the condition resolves.

IMPORTANT SAFETY INFORMATION

Kidney Toxicity and Monitoring: 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: Side effects 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 side effects 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.

What do I do if I have side effects?
Ask your healthcare provider for advice about any side effects that concern you.

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).

The information provided here does not include all that is known about VYONDYS 53. To learn more, talk with your healthcare provider.

Before receiving this infusion, please see the full Prescribing Information for VYONDYS 53 (golodirsen).