Eteplirsen injection

What is this medicine?

Eteplirsen (e TEP ler sun) is a drug which lets dystrophin, the muscle protein missing in Duchenne muscular dystrophy, partially work. This medicine is used for the treatment of Duchenne muscular dystrophy in patients who have a specific gene mutation. It is not a cure.

How should I use this medicine?

This medicine is for infusion into a vein. It is given by a health care professional in a hospital or clinic setting.

Talk to your pediatrician regarding the use of this medicine in children. While this drug may be prescribed for children for selected conditions, precautions do apply.

What side effects may I notice from receiving this medicine?

Side effects that you should report to your doctor or health care professional as soon as possible:

• allergic reactions like skin rash, itching or hives, swelling of the face, lips, or tongue

• signs and symptoms of an infection like fever or chills; cough; sore throat; pain or trouble passing urine

Side effects that usually do not require medical attention (report to your doctor or health care professional if they continue or are bothersome):

• bruising

• dizziness, loss of balance or coordination

• joint pain

• nausea, vomiting

• pain, redness, or irritation at the site where injected

What may interact with this medicine?

Interactions are not expected.

What if I miss a dose?

It is important not to miss your weekly dose. Call your doctor or health care professional if you are unable to keep an appointment.

Where should I keep my medicine?

This drug is given in a hospital or clinic and will not be stored at home.

What should I tell my health care provider before I take this medicine?

They need to know if you have any of these conditions:

• an unusual or allergic reaction to eteplirsen, other medicines, foods, dyes, or preservatives

• pregnant or trying to get pregnant

• breast-feeding

What should I watch for while using this medicine?

Your condition will be monitored carefully while you are receiving this medicine.