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Getting Support

We’re in this together.

When you receive a diagnosis of a rare disease like Duchenne muscular dystrophy, you may feel quite alone in the world. At Sarepta, we want you to know about the wide range of support that’s available to you—from SareptAssist™, our comprehensive patient support program, to national patient advocacy groups that provide education and resources for your entire family.

EXONDYS 51 patient Ricky, age 19, outside in his wheelchair with trees in the background
Meet Ricky, age 19. Deletion of exons 45-50.

SareptAssist: Support, by your side™.

We recognize that families of children receiving treatment for Duchenne muscular dystrophy need more than just medicine. When your child is prescribed EXONDYS 51, we’ll connect you with a dedicated SareptAssist Case Manager who helps with the treatment process. You’ll have support at every step—from managing insurance approvals to coordinating drug delivery. 

Learn How We Can Help
EXONDYS 51 patient Ricky, age 19, outside in his wheelchair with trees in the background
Meet Ricky, age 19. Deletion of exons 45-50.

Connect with the Duchenne community.

Duchenne muscular dystrophy is a rare disease—but you’re not facing it alone. Many families have been in your shoes, and over the years, they’ve created a strong community of patient advocates, educators, and service providers. 

Community
Connect to Community
The Duchenne Community

 

EXONDYS 51 patient Ryan, age 11, in his wheelchair smiling at the camera

Resources on Duchenne.

Find educational resources that may be of interest to you.

Explore Resources
EXONDYS 51 patient Ryan, age 11, in his wheelchair smiling at the camera

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).

READ MORE

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).