Durveqtix%252525252525252525252525252525252525252525252525252525252525252525252525252520gene%252525252525252525252525252525252525252525252525252525252525252525252525252520therapy

# Durveqtix gene therapy ## Overview Durveqtix is a gene therapy used for the treatment of adults and children 12 years of age and older with Duchenne muscular dystrophy (DMD) who are able to produce dystrophin protein. Durveqtix is a one-time intravenous infusion. ## Primary Indications Treatment of Duchenne muscular dystrophy (DMD) in patients 12 years of age and older who are able to produce dystrophin via the microdystrophin gene, as confirmed by genetic testing. ## Adult Dosing A single intravenous infusion of 2.5 x 10^13 vg/kg. ## Pediatric Dosing A single intravenous infusion of 2.5 x 10^13 vg/kg for patients 12 years of age and older. Dosing for pediatric patients younger than 12 years has not been established. ## Dose Adjustments No dose adjustments are specified for renal or hepatic impairment. ## Contraindications None are listed on the prescribing information. ## Adverse Effects Common adverse effects include pyrexia, platelet count decrease, vomiting, alanine aminotransferase increase, aspartate aminotransferase increase, creatine kinase increase, troponin-I increase, and fatigue. Serious adverse effects may include hypersensitivity reactions and potential for liver injury. ## Key Drug Interactions The potential for drug interactions has not been fully evaluated. Caution should be exercised when co-administering with other medications known to cause liver injury. ## Monitoring Monitor for signs and symptoms of hypersensitivity reactions during and after infusion. Liver function tests (ALT, AST, bilirubin) should be monitored prior to infusion and at regular intervals for at least 3 months post-infusion. Monitor platelet counts. Patients should be monitored for cardiac abnormalities. ## Clinical Pearls Durveqtix is a one-time treatment and should be administered by a healthcare professional experienced in gene therapy. Premedication with antipyretics and corticosteroids may be considered to mitigate infusion-related reactions. Patients should remain under observation for at least 2 hours post-infusion. There is a risk of developing antibodies to the AAV capsid. *** **Disclaimer:** This information is intended for healthcare professionals and is not a substitute for professional medical advice. Always consult the most current prescribing information and your healthcare provider for any questions or before making any decisions related to patient care.