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Exondys 51

Generic Name: eteplirsen (e TEP lir sen)
Brand Name: Exondys 51
Physician reviewed Exondys 51 patient information - includes Exondys 51 description, dosage and directions.
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Drug Information:
Exondys 51 works by helping the body produce a protein called dystrophin. Dystrophin is a protein that helps stabilize the structure of muscle tissue and is necessary for proper muscle development and function. Without dystrophin, muscle fibers can become weak and easily damaged. This can affect voluntary movement such as walking. Children with Duchenne muscular dystrophy have a mutation of a gene that would normally cause the body to produce dystrophin. Exondys 51 is used in children with Duchenne muscular dystrophy (DMD). This medicine is not a cure for DMD, but Exondys 51 may lessen muscle weakness and muscle wasting caused by this disease. Learn more

Exondys 51 Side Effects

Exondys 51 Side Effects

Note: This document contains side effect information about eteplirsen. Some of the dosage forms listed on this page may not apply to the brand name Exondys 51.

For the Consumer

Applies to eteplirsen: intravenous solution

Along with its needed effects, eteplirsen (the active ingredient contained in Exondys 51) may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur while taking eteplirsen:

More common

  • Loss of balance control
  • vomiting

Some side effects of eteplirsen may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

Incidence not known

  • Bleeding, blistering, burning, coldness, discoloration of the skin, feeling of pressure, hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site
  • blistering, burning, crusting, dryness, or flaking of the skin
  • itching, scaling, severe redness, soreness, or swelling of the skin

For Healthcare Professionals

Applies to eteplirsen: intravenous solution


The more commonly reported adverse events have included balance disorder and vomiting.


Very common (10% or more): Upper respiratory tract infection


Frequency not reported: Hypersensitivity reactions including rash, urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, hypotension


Very common (10% or more): Contact dermatitis (25%), contusion, facial flushing, transient erythema, excoriation, rash


Very common (10% or more): Vomiting (38%)


Very common (10% or more): Catheter site pain


Very common (10% or more): Arthralgia

Nervous system

Very common (10% or more): Balance disorder (38%)

Editorial References and Review

Medically reviewed by BestRx Medical Team Last updated on 1/1/2020.

Source: Drugs.com Exondys 51