Building on hope for Duchenne for those amenable to exon 53 skipping

Meet Elijah, age 8
Amenable to exon 53 skipping

Meet Nicholas, age 17
Amenable to exon 53 skipping

Meet Luke, age 12
Amenable to exon 53 skipping

Meet Andres, age 9
Amenable to exon 53 skipping

VYONDYS 53 is an FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 53. In some patients, it helps the body make a shorter form of the dystrophin protein.

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. VYONDYS 53 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for VYONDYS 53 to continue to be approved.

Discover VYONDYS 53

Who Can Take It?

Learn the importance of understanding your genetic mutation and what it means to be amenable to treatment with VYONDYS 53

Learn About Amenability

Starting Treatment

Starting treatment with VYONDYS 53? Let us take you through the process so you know what to expect.

Explore the Process

Getting Support

With the SareptAssist patient support program, we'll help you navigate the process of starting therapy with VYONDYS 53.

How We Can Help

“I’m glad to have “SareptAssist” as a resource for our family.”

Liz, Luke’s mom

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).