Eteplirsen
Mechanism :
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.
Indication :
- Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
Contraindications :
No contraindications.
Dosing :
Male children ≥7 years: IV 30 mg/kg/dose once weekly.
Adverse Effect :
Equilibrium disturbance, contact dermatitis, vomiting, bronchospasm, cough, dyspnea.
Interaction :
No known significant interactions.
Hepatic Dose :
No dosage adjustments are recommended.