Supporting you throughout your journey.

During your VYONDYS 53 treatment journey, it’s important to stay informed. We’re committed to keeping you updated with information on treatment, education, and support.

Sign Up for Updates

Meet Nicholas, age 17
Amenable to exon 53 skipping

 


Support, by your side
 

SareptaAssist

 


SareptAssist is designed to provide the information you need to navigate the process of starting and staying on therapy. Your Case Manager will work closely with you to provide one-on-one, personalized support.
 

Learn About SareptAssist

 

 


Connect with community.
 


The Duchenne muscular dystrophy community is informative, understanding, and well connected. And they are ready to help with the kind of support that comes only from people who know what you’re going through.
 

Find Community Resources

 

 

 

Related FAQs

Will my insurance cover VYONDYS 53?

Once your child is prescribed VYONDYS 53, you may enroll in SareptAssist, our patient support program. Your Case Manager will start a benefits investigation of your current insurance plan and can help explain details about your coverage. How SareptAssist can help.

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

 

 

Desktop star

Sign up to get updates
from Sarepta.

Continue

 

 

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.

Read More

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).