What is Golodirsen?
Golodirsen can be used to treat Duchenne muscle dystrophy in both adults and children with a specific gene mutation. You will be tested by your doctor for this mutation.The FDA approved Golodirsen on an "accelerated basis". Some people have responded to golodirsen in clinical studies. However, further research is needed.Golodirsen can be used in other ways not mentioned in this guide.
Side effects of Golodirsen
If you experience any of the following symptoms of an allergic reaction, seek immediate medical attention: hives; itching; rash; blistering or peeling skin; fever.
Golodirsen may cause serious side effects. If you experience:
- Pink, brown, or red urine
- Foaming urine
- You may experience swelling of the face, hands, or feet.
Some of the common side effects associated with golodirsen include:
- Headache;
- Fever;
- Stomach pain;
- Falls;
- Runny or stuffy nasal symptoms
There may be other side effects. For medical advice on side effects, call your doctor. Contact the FDA by dialing 1-800-FDA-1088 in order to report side effects.
Similar/related drugs
Emflaza, Elevidys, deflazacort, Viltepso, delandistrogene moxeparvovec, eteplirsen
Warnings
Please follow all of the directions outlined on your medication's package or label carefully before beginning to take it. Inform your healthcare provider about your medical conditions, allergies, and medicines.
Before you take this drug
Inform your doctor if kidney problems have ever occurred.Inform your physician immediately if you become pregnant or nursing.
How to take Golodirsen?
You will be tested by your doctor to ensure that you are not suffering from any conditions that would make it unsafe for you to use golodirsen.In general, Golodirsen infusions are given once a week. This injection will be given by a healthcare provider.The infusion may take 60 minutes.You may need to have your kidney function checked if you use this medication.
Details on dosage
Adult dose for muscle dystrophy:
30 mg/kg via IV infusion weekly
Comments:
The approval of this indication will be contingent on the confirmation of clinical benefit through confirmatory studies.
Use: To treat Duchenne muscle dystrophy in patients who have a mutation of the DMD genes that can be treated by exon 53-skipping.
The usual paediatric dose for muscular dystrophy:
30 mg/kg via IV infusion weekly
Comments:
The approval of this indication will be contingent on the confirmation of clinical benefit through confirmatory studies.
Use: To treat Duchenne muscular dystrophy (DMD) in patients who have a mutation of the DMD genes that allows exon 53 to be skipped.
What happens if I miss the dose?
If you are unable to attend your scheduled golodirsen treatment, call your doctor.
What happens if I overdose?
Call 1-800-222-1222 immediately if you suspect poisoning and require immediate assistance or seek immediate medical treatment.
What should be avoided?
If your doctor prescribes any restrictions regarding food, beverage, or activity, follow their instructions.
Interaction with other drug
Other medications, such as vitamins and herbal products, may also affect golodirsen. Inform your doctor of all the medicines you are taking and those that you stop or start using.
