Golodirsen Medication

Golodirsen (Vyondys 53) is used for the treatment of patients with DMD (Duchenne muscular dystrophy) in those who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Dosage: The recommended golodirsen dose is 30 mg/kg, which should be administered once weekly as 35 to 60 minute by intravenous infusion. In occurrence of a missed dose of golodirsen, it should be administered as soon as possible following the scheduled dose.

Side Effects: The most commonly reported vyondys side effects include:

  • Headache
  • Pyrexia
  • Fall
  • Cough
  • Vomiting
  • Nausea
  • Abdominal pain
  • Nasopharyngitis

Warnings and Precautions:

  • Patients receiving golodirsen 100 mg injection with known renal function impairment should be closely monitored while on therapy.
  • Measure patients for glomerular filtration rate prior to the initiation of vyondys 100 mg injection therapy.
  • Patients taking treatment with the drug named vyondys 53 are advised not to use this therapy concomitantly with other exon skipping therapies for DMD.
  • The vyondys 53 uses could cause headache in patients. If you have a headache while taking this drug, talk with your healthcare team.