Medically reviewed on Oct 31, 2018
Commonly used brand name(s)
In the U.S.
- Exondys 51
Available Dosage Forms:
Therapeutic Class: Musculoskeletal Agent
Uses For eteplirsen
Eteplirsen injection is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact.
Eteplirsen is to be given only by or under the direct supervision of a doctor.
Before Using eteplirsen
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For eteplirsen, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to eteplirsen or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of eteplirsen injection in children.
No information is available on the relationship of age to the effects of eteplirsen injection in geriatric patients.
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Interactions with Medicines
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Interactions with Food/Tobacco/Alcohol
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Proper Use of eteplirsen
A nurse or other trained health professional will give you eteplirsen in a hospital. Eteplirsen is given through a needle placed in one of your veins.
Precautions While Using eteplirsen
Your doctor will check your progress closely while you are receiving eteplirsen. This will allow your doctor to see if the medicine is working properly and to decide if you should continue to receive it.
Eteplirsen Side Effects
Along with its needed effects, a medicine 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:
- Loss of balance control
Some side effects 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
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
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More about eteplirsen
- Eteplirsen Side Effects
- During Pregnancy
- Dosage Information
- En Español
- Drug class: miscellaneous uncategorized agents
Other brands: Exondys 51