US approves gene therapy treatment for hemophilia
Washington (AFP) - Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday. Beqvez, which is given as a single intravenous infusion, was shown in a clinical trial of 45 people to be better at preventing bleeding among adults with moderate to severe hemophilia B compared to regular infusions of a protein that promotes clotting, called protein factor IX (FIX). The current standard of care is cumbersome, requiring infusions up to several times per week. "Many people with hemophilia ...