Miscellaneous musculoskeletal disorders:
Indications for VILTEPSO:
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 60mins. 80mg/kg once weekly.
Warnings/Precautions:
Monitor renal function. Measure baseline serum cystatin C, urine dipstick, urine protein-to-creatinine ratio, and consider measuring GFR prior to initiation. During treatment, monitor urine dipstick monthly, serum cystatin C and urine protein-to-creatinine ratio every 3 months. Refer to pediatric nephrologist if a persistent increase in serum cystatin C or proteinuria is detected. Renal impairment: monitor closely. Pregnancy. Nursing mothers.
Pharmacologic Class:
Antisense oligonucleotide.
Interactions:
Do not mix or infuse with other drugs concomitantly via same IV access line.
Adverse Reactions:
Upper respiratory tract infection, inj site reaction, cough, pyrexia; renal toxicity.
Generic Availability:
NO
How Supplied:
Single-dose vial (5mL)—1
