About Golodirsen
Class: | Antisense oligonucleotide
Use: | Golodirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Adult dose: | The standard adult dosing is not established as Golodirsen is primarily used in pediatric populations.
Pediatric dose: | The recommended dose is 30 mg/kg once weekly as an intravenous infusion.
Side effects: | Common side effects include headache, fever, falls, abdomin
Drug Class
Antisense oligonucleotide
Uses & Indications
Golodirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Storage Requirements
Store Golodirsen vials in a refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light.
Manufacturer & Packaging
Manufacturer: Sarepta Therapeutics Inc., United States of America
Package Size
2ml Vial
Price & Supplier
Price in UAE: AED 7158.03
Dosage Information
Adult Dose
The standard adult dosing is not established as Golodirsen is primarily used in pediatric populations.
Pediatric Dose
The recommended dose is 30 mg/kg once weekly as an intravenous infusion.
Side Effects
Common side effects include headache, fever, falls, abdominal pain, nasopharyngitis, cough, vomiting, and nausea.
Contraindications & Precautions
There are no known contraindications for Golodirsen. However, it should be used with caution in patients with known hypersensitivity to the drug or its components.
Important Warnings
Monitor renal function as Golodirsen may cause kidney toxicity. Regular urine tests and blood tests are recommended to assess renal function during treatment.
Medical Disclaimer
This information is for educational purposes only and should not replace professional medical advice.
Always consult with a licensed healthcare provider or pharmacist in the UAE before taking any medication.
The information presented here is specific to medicines available in UAE pharmacies and is regularly updated
to ensure accuracy for patients and healthcare professionals in the United Arab Emirates.