What is Exondys 51? Uses, warnings & side effects

Updated May 14, 2025 • Published Dec 30, 2020

Exondys 51 overview

BRAND NAMES:

Exondys 51

Generic Names:

Exondys 51

Uses:

Treats Duchenne muscular dystrophy.

Therapeutic Classes:

Musculoskeletal Agent

Forms:

Injectable

How it’s taken:

By injection

What is Exondys 51 used for?

Exondys 51 is used in the treatment of duchenne muscular dystrophy (DMD) in patients who have a DMD gene mutation and is responsive to exon 51 skipping. It increases the levels of dystrophin in the skeletal muscles of patients with this condition. Dystrophin prevents muscles from becoming weak and easily damaged. It is indicated for use in adults and children. This medication is available as an injection.

What form(s) does Exondys 51 come in?

Injectable

What are common Exondys 51 dosages?

Vial 2ML of 100MG/2ML
Vial 10ML of 500MG/10ML

Exondys 51 images - What does Exondys 51 look like?

Exondys 51
10ml of 500mg/10ml vial
Exondys 51
2ml of 100mg/2ml vial

How to take Exondys 51

Injection routeYour doctor will prescribe your dose and schedule. This medicine is given through a needle placed in a vein.
Injection routeA nurse or other health provider will give you this medicine.
Injection routeYour doctor will prescribe your dose and schedule. This medicine is given through a needle placed in a vein.
Injection routeA nurse or other health provider will give you this medicine.

What to do if you miss a dose of Exondys 51

Exondys 51 contraindications - Who shouldn't take Exondys 51?

This medicine is not right for everyone. You should not receive it if you had an allergic reaction to eteplirsen.

Exondys 51 warnings

Tell your doctor if you are pregnant or breastfeeding, or if you have kidney disease or liver disease.
Your doctor will check your progress and the effects of this medicine at regular visits. Keep all appointments.

Exondys 51 interactions & what to avoid when taking Exondys 51

Exondys 51 side effects - How does Exondys 51 make you feel?

Serious Side Effects

Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
Unusual bruising
Blistering, peeling, red skin rash
Fever, chills, cough, sore throat, body aches

Less Serious Side Effects

Pain, itching, burning, swelling, or a lump under your skin where the needle is placed
Problems with balance
Joint pain
Vomiting