What is this medicine?
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.
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 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 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):
What may interact with this medicine?
Interactions are not expected.
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.
Where should I keep my medicine?
This medicine 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 golodirsen, other medicines, foods, dyes, or preservatives
pregnant or trying to get pregnant
What should I watch for while using this medicine?
Your condition will be monitored carefully while you are receiving this medicine.