Leap forward towards gene therapy cure for haemophilia A

Scientists around the world congratulate the team that has made a major advance in finding a cure for the life-threatening blood disorder

Scientists believe they are on the way to finding a cure for haemophilia A, the bleeding disorder that currently requires sufferers to inject themselves every other day to avoid life-threatening complications.

One dose of a gene therapy given experimentally to 13 patients by NHS doctors in the UK has allowed them all to come off treatment. These were men – most sufferers are – who would not only bleed without stopping from an injury but would bleed into their joints even in their sleep causing pain and disability, without frequent injections of a clotting factor. None of them now bleeds spontaneously in that way.

Continue reading... December 14, 2017 at 05:31AM