Creating a treatment plan that works.

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Starting treatment for Duchenne may feel like entering unknown territory. By creating a treatment plan up front, you can help make the experience more comfortable for your child and less stressful for you. Let’s talk about a few options you should discuss with your doctor when creating a treatment plan that will work for your family. And remember, your SareptAssist Case Manager is ready to provide information on any questions that come up.

 

Questions?

Give us a call.
 


1-888-SAREPTA

1-888-727-3782
Case Managers are available Monday – Friday
8:30 am – 6:30 pm ET

 

Where to get treatment.

VYONDYS 53 treatment is a weekly infusion that is always given by a trained healthcare provider. You may be wondering how that works. Depending on your doctor’s instructions, your insurance plan coverage, and your own preferences, your child’s infusions will take place in one of these locations:

 

At an infusion center.

A medical clinic that specializes in infusions. These facilities provide infusion services for a variety of medications and are staffed with nurses and technicians who are well versed in performing infusions. 

At home.

Performed by an experienced nurse. You may have the option of in-home infusions, where the nurse comes to you and your child can remain at home. Ask your child’s doctor for a recommendation.

At a doctor’s office.

Medical facilities equipped to offer infusions. Your doctor’s office or local hospital may be equipped to perform outpatient infusions. Talk to your doctor about what they recommend.

Considerations:
 

  • Your doctor’s recommendation. What does your doctor say about infusing at a center vs infusing at home?
  • Time and convenience. You’ll need to block out time for your infusion. Call ahead to ask your nurse or doctor what to expect.
  • Coverage. Every insurance plan is different, so be sure to connect with your SareptAssist Case Manager if you have questions about which infusion locations are covered by your plan.
Nicholas, age 17 Amenable to exon 53 skipping

When to schedule treatment.

It’s important to keep your therapy schedule as consistent as possible. Try to choose a day and time that you’ll be able to keep, week in and week out.

What about vacation?

Let your Case Manager know if you’re planning a trip. There may be an option to arrange an infusion at your destination to keep your treatment schedule on track. 

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How to choose: An IV or a port?

VYONDYS 53 is given by a weekly intravenous (IV) infusion, which is a method of delivering medicine directly into your bloodstream through a vein. This is typically done through a temporary IV that is inserted before and removed after treatment, or through a port, which is implanted in the chest or arm and remains in the body. Discuss your options with your child's doctor.

 

Next: Learn what to expect on infusion day. 
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Nicholas, age 17 Amenable to exon 53 skipping – with his family and dog

“I’m grateful for SareptAssist’s
help when I have questions
about the process.”

-Erin, Nicholas’ mother

Related FAQs

What happens if I miss an infusion?

Talk to your healthcare provider. If you miss a dose of VYONDYS 53, it may be administered as soon as possible after the scheduled time.

What is SareptAssist?

SareptAssist is our patient support program. Families are connected with dedicated Case Managers who provide information and support to help them start and stay on treatment with VYONDYS 53. How SareptAssist can help.

See All FAQs

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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.

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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.

 

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).