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.
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