Miscellaneous musculoskeletal disorders:
Indications for VYONDYS 53:
Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Adults and Children:
Give as IV infusion over 35–60mins. 30mg/kg once weekly.
Measure glomerular filtration rate (GFR) prior to initiation. Monitor proteinuria monthly and serum cystatin C every 3 months; if confirmed dipstick proteinuria of ≥2+ or elevated serum cystatin C, perform 24-hr urine collection. Monitor renal function during therapy. Interrupt or slow infusion if hypersensitivity reaction occurs. Renal impairment: monitor closely. Pregnancy. Nursing mothers.
Do not mix or dilute with other medications.
Headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, nausea; renal toxicity, hypersensitivity reactions.
Single-dose vial (2mL)—1