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

Pricing for VILTEPSO

5ml of 50mg/ml vial (Qty: 1)
Appx. price $297
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