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Talking to your doctor about EXONDYS 51 (eteplirsen).

Understanding the treatment possibilities for your child’s Duchenne involves talking with their doctor about genetic testing and what the test results mean. You’ll also want to discuss your child’s mutation and whether it is amenable to exon-skipping therapy.

Use our step-by-step tool to create a personalized Doctor Discussion Guide with the suggested questions to ask at your child’s next appointment.

  • Learn about tests required to diagnose Duchenne muscular dystrophy.
  • Understand genetic test results and your child’s mutation.
  • Find out the importance of when your genetic test was performed.
  • Get a guide that’s customized for you.

Just answer a few simple questions to get started!

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Discussion guide
Has your child received a diagnosis of Duchenne muscular dystrophy?
Discussion guide

Talk with your child’s doctor about CK testing.

If you suspect that your child has Duchenne muscular dystrophy, ask your child's doctor about taking a creatine kinase (CK) test. Be prepared to discuss your child’s development and any symptoms you’ve observed. If your child’s doctor suspects a muscle disease, they may recommend the CK test—a simple, inexpensive blood test that measures the amount of creatine kinase in the blood. Elevated CK levels may indicate muscle damage.

If your child’s test results show elevated CK levels, find out if the next appropriate step is genetic testing to confirm a Duchenne diagnosis and identify your child’s mutation. Decode Duchenne offers free genetic testing to the Duchenne community and can provide genetic counselors to interpret test results.

Visit Decode Duchenne to learn more.

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Discussion guide
How was your child diagnosed with Duchenne?
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Discussion guide
How long ago was the genetic test that confirmed a Duchenne diagnosis performed?
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Discussion guide
Did you understand your child’s genetic test results?
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Discussion guide
Do you know your child’s mutation?
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Discussion guide
Have you discussed treatment options with your child’s doctor?
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Discussion guide
Have you discussed genetic testing or treatment options with your child’s doctor?
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Discussion guide

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WHAT IS EXONDYS 51® (eteplirsen)?

EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51.

EXONDYS 51 was 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 that has not been proven. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

In patients who received EXONDYS 51 for up to 4 years in clinical studies, the following side effects were reported in at least 10% of patients and occurred more often than at the same dose of EXONDYS 51: vomiting, bruising, scratches, joint pain, rash, IV site pain, and infections of the upper airways.

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).

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WHAT IS EXONDYS 51® (eteplirsen)?

EXONDYS 51 is used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51.

EXONDYS 51 was 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 that has not been proven. EXONDYS 51 treatment increased the marker, dystrophin, in skeletal muscle in some patients. Verification of a clinical benefit may be needed for EXONDYS 51 to continue to be approved.

IMPORTANT RISK INFORMATION

Allergic reactions, including rash, hives, fever, flushing, cough, difficulty breathing, wheezing, and low blood pressure have occurred in patients who were treated with EXONDYS 51. Seek immediate medical care if signs and symptoms of allergic reactions occur.

Side effects that happened at least 25% more often in 8 patients treated with EXONDYS 51 by intravenous infusion than in 4 patients treated with an inactive intravenous infusion were problems with balance (38%, 0%), vomiting (38%, 0%), and skin irritation (25%, 0%). The most common side effects were problems with balance and vomiting.

In patients who received EXONDYS 51 for up to 4 years in clinical studies, the following side effects were reported in at least 10% of patients and occurred more often than at the same dose of EXONDYS 51: vomiting, bruising, scratches, joint pain, rash, IV site pain, and infections of the upper airways.

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 EXONDYS 51 (eteplirsen).