Genetic Implications: Genetic Implications


Trade Name(s)

  • Vyondys 53

Ther. Class.

none assigned

Pharm. Class.

antisense oligonucleotides


Genetic implication  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)



Contraindicated in:

  • None reported.

Use Cautiously in:

  • Renal impairment;
  • OB:   Safety not established in pregnancy
  • Lactation:  Safety not established in breastfeeding.

Adverse Reactions/Side Effects

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.



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.


  • May apply topical anesthetic cream to infusion site before infusion.
  • Intermittent Infusion:  Allow vials to warm to room temperature. Mix contents of each vial by inverting gently 2–3 times; do not shake. Solution is clear to slightly opalescent, colorless, and may contain trace amounts of small, white to off-white amorphous particles; do not administer solutions that are cloudy, discolored, or contain additional particulate matter. Withdraw volume required from each vial with 21–gauge or smaller needle. Dilution:  0.9% NaCl for total volume of 100–150 mL. Gently invert 2–3 times to mix; do not shake. Solution is stable for 4 hr at room temperature including infusion time or 24 hr if refrigerated. Do not freeze. Discard unused solution.
  • Rate: Infuse over 35–60 min via an in-line 0.2 micron filter. Flush line with 0.9% NaCl before and after infusion.
  • Y-Site Incompatibility: Do not administer with or mix with other medications.

Patient/Family Teaching

  • Explain purpose of medication to patient.
  • Advise patient to notify health care professional immediately if signs and symptoms of hypersensitivity reactions occur.
  • Advise patient to notify health care professional if pregnancy is planned or suspected or if breastfeeding.
  • Emphasize importance of regular lab tests to monitor for renal toxicity.

Evaluation/Desired Outcomes

Increased dystrophin production.