• Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Contraindicated in:

• None reported.

Use Cautiously in:

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

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.

Drug-Drug:

• None reported.

• Solution for injection: 50 mg/mL;

• IV (Adults and Children): 30 mg/kg once weekly.

Vyondys 53

• 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.

Therapeutic Classification: n/a

Pharmacologic Classification: antisense oligonucleotides

Absorption: IV administration results in complete bioavailability.

Distribution: Widely distributed to extravascular tissues.

Metabolism/Excretion: Not hepatically metabolized. Primarily excreted unchanged in urine.

Half-life: 3.4 hr.

(plasma concentrations)

• 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.

GOE-loe-DIR-sen