- Exondys 51
temporary class antisense oligonucleotides
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
Adverse Reactions/Side Effects
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
- Monitor for signs and symptoms of hypersensitivity reactions (rash, urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, hypotension) during infusion. Treat symptomatically. Consider slowing or interrupting infusion.
- Activity intolerance (Indications)
- Apply topical anesthetic cream to infusion site prior to infusion. Flush IV line with 0.9% NaCl before and after infusion.
- Intermittent Infusion: Allow vials to reach room temperature before using; mix by inverting 2–3 times, do not shake. Solution is clear and colorless with some opalescence; do not administer solutions that are discolored or contain particulate matter. Diluent: Withdraw dose from vials and dilute with 0.9% NaCl for a total volume of 100–150 mL. Complete infusion within 4 hrs. Diluted solution stable for up to 24 hrs if refrigerated. Do not freeze.
- Rate:Infuse over 35 to 60 min.
- Y-Site Incompatibility: Do not infuse other medication concomitantly in same IV access line with eteplirsen.
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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