Resources and Events

Meet Colin, age 11. Amenable to exon 45 skipping

For your information.

Learn. Connect. Get involved. If you’re ready to expand your knowledge of Duchenne muscular dystrophy or connect with the community face-to-face, you can get started here.

Duchenne educational resources.

Getting Started on AMONDYS 45. This guide can help you understand more about Duchenne, how exon skipping works, and how to get started on AMONDYS 45.

Download Brochure (PDF)

Duchenne community events.

Duchenne advocacy organizations often sponsor or participate in events that may be of interest to you. Find out more through the organizations listed below.

From Family and Caregiver Workshops to sporting events and fundraisers, Cure Duchenne provides ample opportunity to connect with others in the Duchenne community.

Learn more about Cure Duchenne events.

Jett Foundation empowers people and families impacted by Duchenne muscular dystrophy through the development of transformative programming, educational opportunities, and ongoing support for every stage of a Duchenne journey.

Learn more about JETT Foundation events.

The Muscular Dystrophy Association offers families and caregivers several ways to get involved, from Muscle Walks to fundraising events.

Learn more about MDA events.

Parent Project Muscular Dystrophy hosts events that provide opportunities for families, caregivers, and those living with Duchenne to gather, build support networks, and discuss all topics Duchenne.

Learn more about PPMD events.

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Related FAQs

Who can take AMONDYS 45?

Duchenne patients who receive AMONDYS 45 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 45. Your child's doctor is best equipped to determine if your child’s mutation is amenable to treatment with AMONDYS 45. We’ve developed a Doctor Discussion Guide to help you start that important conversation.

What support is available for people being treated with AMONDYS 45?

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. See how to get started.

See All FAQs

What is AMONDYS 45 (casimersen)?

AMONDYS 45 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the dystrophin gene that can be treated by skipping exon 45.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Contraindications: Do not receive AMONDYS 45 if you are allergic to casimersen or any of the ingredients in AMONDYS 45. Serious allergic reactions to casimersen have included anaphylaxis, which may include difficulty breathing, tightness in the chest, and angioedema which may include swelling of the mouth, face, lips, or tongue.

Hypersensitivity Reactions: Serious allergic reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. Patients should seek immediate medical care should they experience signs and symptoms of allergic reactions. Your doctor will institute appropriate medical treatment which may include slowing, interrupting, or discontinuing the AMONDYS 45 infusion. Your doctor will monitor you until the condition resolves.

Kidney Toxicity and Monitoring: Damage to the kidneys was seen in animals who received casimersen. Although damage to the kidneys was not seen in clinical studies with AMONDYS 45, 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 occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in patients who received an inactive intravenous (IV) infusion were (AMONDYS 45, placebo): upper respiratory tract infection (65%, 55%), cough (33%, 26%), fever (33%, 23%), headache (32%, 19%), joint pain (21%, 10%), and pain in mouth and throat (21%, 7%).

Other side effects that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than patients who received an inactive IV infusion were: ear pain, nausea, ear infection, pain after injury, and dizziness and light-headedness.

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 AMONDYS 45. To learn more, talk with your healthcare provider.

Before receiving this infusion, please see the full Prescribing Information for AMONDYS 45 (casimersen).