Blindness Cure Is Out of Sight
by Craig Klugman, Ph.D.
The FDA has approved the world’s first gene therapy: Luxturna (voretigene neparvovec; AAV2-hRPE65v2) is a one-time intervention that can treat an inherited retinal disease (RPE65-mediated inherited retinal dystrophy). The disease is rare, affecting only 1,000-2,000 people in the U.S. The treatment works by introducing a virus to the eye that contains a healthy copy of the gene that caused the blindness. In a phase 3 trial of 31 subjects (20 intervention; 9 control; 2 withdrew), 65% of patients who received the procedure experienced dramatic improvements in their vision.…
Source: blog.bioethics.net - Category: Medical Ethics Authors: Craig Klugman Tags: Clinical Trials & Studies Featured Posts Health Disparities Health Policy & Insurance Health Regulation & Law Pharmaceuticals gene therapy Luxturna Source Type: blogs
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