Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
Binds to exon 51 of dystrophin pre-messenger RNA (mRNA), resulting in exclusion of this exon during mRNA processing. Exon skipping allows for production of an internally truncated dystrophin protein.
Increased dystrophin production.
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism and Excretion: Not hepatically metabolized; excreted renally.
Half-life: 3–4 hr.
TIME/ACTION PROFILE (plasma concentrations)
Use Cautiously in:
OB: Lactation: Safety and effectiveness not established
Neuro: balance disorder
Resp: upper respiratory tract infection
Misc: HYPERSENSITIVITY REACTIONS (INCLUDING RASH, URTICARIA, PYREXIA, FLUSHING, DYSPNEA, BRONCHOSPASM, AND HYPOTENSION)
* CAPITALS indicate life-threatening.
Underline indicate most frequent.
IV Children: 30 mg/kg once weekly.
Solution for IV infusion (must be diluted): 50 mg/mL
Instruct patient and parents to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.
Increase in dystrophin levels in muscle tissues.
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