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.

Pharmacologic Class:

Antisense oligonucleotide.


Do not mix or dilute with other medications.

Adverse Reactions:

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

Generic Availability:


How Supplied:

Single-dose vial (2mL)—1

Pricing for VYONDYS 53

2ml of 50mg/ml vial (Qty: 1)
Appx. price $1642