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 gene therapies secured for NHS patients at an affordable price and becomes the first drug to be made available in our Innovative Medicines Fund to provide early access for patients while further data is collected on its long-term benefits.”…
