UK – Responding to new final draft guidance from NICE for a new gene therapy for haemophilia B

Professor Sir Stephen Powis, NHS National Medical Director, said: “This transformative gene therapy is the first of its kind for haemophilia B patients on the NHS and has the potential to significantly improve the lives of hundreds of people by helping to reduce symptoms such as painful bleeds. “It is a one-time therapy that could be truly life-changing for some, as it could help people avoid the need for regular hospital visits. “This promising drug is the latest in a series of pioneering g...