Miscellaneous musculoskeletal disorders:

Indications for: VYONDYS 53

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.

VYONDYS 53 Warnings/Precautions:

Monitor renal function. Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio, and consider measuring GFR prior to initiation. During treatment, monitor urine dipstick monthly, and serum cystatin C and urine protein-to-creatinine ratio every 3 months. To test urine protein: may use urine samples obtained prior to infusion or at least 48hrs after the most recent infusion. Refer to a pediatric nephrologist if a persistent increase in serum cystatin C or proteinuria is detected. Interrupt or slow infusion if hypersensitivity reaction occurs. Renal impairment: monitor closely. Pregnancy. Nursing mothers.

VYONDYS 53 Classification:

Antisense oligonucleotide.

VYONDYS 53 Interactions:

Do not mix or infuse with other drugs concomitantly via same IV access line. May cause false (+) results for urine protein with using pyrogallol red reagent.

Adverse Reactions:

Headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, nausea; renal toxicity, hypersensitivity reactions.

Generic Drug Availability:


How Supplied:

Single-dose vial (2mL)—1