Golodirsen injection

What is this medication?

GOLODIRSEN (GOE loe DIR sen) 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.

This medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

COMMON BRAND NAME(S): Vyondys

What should I tell my care team before I take this medication?

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

• kidney disease
• an unusual or allergic reaction to golodirsen, other medicines, foods, dyes, or preservatives
• pregnant or trying to get pregnant
• breast-feeding

How should I use this medication?

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 selected conditions, precautions do apply.

Overdosage: If you think you have taken too much of this medicine contact a poison control center or emergency room at once.

NOTE: This medicine is only for you. Do not share this medicine with others.

What if I miss a dose?

Keep appointments for follow-up doses. It is important not to miss your dose. Call your doctor or healthcare professional if you are unable to keep an appointment.

What may interact with this medication?

Interactions are not expected.

This list may not describe all possible interactions. Give your health care provider a list of all the medicines, herbs, non-prescription drugs, or dietary supplements you use. Also tell them if you smoke, drink alcohol, or use illegal drugs. Some items may interact with your medicine.

What should I watch for while using this medication?

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

What side effects may I notice from receiving this medication?

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 kidney injury like trouble passing urine or change in the amount of urine

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

• cough
• fever
• headache
• nausea, vomiting
• stomach pain

This list may not describe all possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Where should I keep my medication?

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

NOTE: This sheet is a summary. It may not cover all possible information. If you have questions about this medicine, talk to your doctor, pharmacist, or health care provider.