Genetic Implications:
Pronunciation:
goe-loe-dir-sen
Trade Name(s)
Ther. Class.
none assigned
Pharm. Class.
antisense oligonucleotides
Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 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.
Therapeutic Effect(s):
Increased dystrophin production.
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed to extravascular tissues.
Metabolism and Excretion: Not hepatically metabolized. Primarily excreted unchanged in urine.
Half-life: 3.4 hr.
TIME/ACTION PROFILE (plasma concentrations)
ROUTE | ONSET | PEAK | DURATION |
---|---|---|---|
IV | unknown | unknown | unknown |
Contraindicated in:
Use Cautiously in:
CV: tachycardia
EENT: nasopharyngitis
GI: abdominal pain, nausea, vomiting, constipation, diarrhea
GU: nephrotoxicity
Local: injection site pain
MS: back pain
Neuro: headache, dizziness
Resp: cough
Misc: falls, fever, hypersensitivity reactions
* CAPITALS indicate life-threatening.
Underline indicate most frequent.
Drug-Drug
None reported.
IV (Adults and Children): 30 mg/kg once weekly.
Solution for injection: 50 mg/mL
Monitor for signs and symptoms of hypersensitivity reactions (rash, fever, pruritus, urticaria, dermatitis, skin exfoliation) during infusion. If hypersensitivity reaction occurs, consider slowing infusion rate or interrupting therapy. Treat symptomatically.
Lab Test Considerations:
Assess serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio before starting therapy. Monitor glomerular filtration rate (GFR) by 24-hr urine collection before starting and periodically during therapy; serum creatinine may not be a reliable measure due to reduced muscle mass. Monitor for proteinuria by dipstick urinalysis monthly. Monitor serum cystatin C and urine protein-to-creatinine ratio every 3 mo. Use urine obtained on the day of infusion prior to infusion or urine obtained at least 48 hr after most recent infusion. If proteinuria or persistent elevated serum cystatin C occurs, refer to pediatric nephrologist for further evaluation.
Advise patient to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.
Increased dystrophin production.