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Haemophilia A gene therapy results 'mind-blowing'

14-Dec-2017

Key points from article :

Haemophilia prevents production of a protein needed to stop bleeding.

Even the impact of walking can lead to bleeding in joints.

Sufferers typically need injections of factor VIII every other day for life.

Trial resulted in 11 of 13 participants producing near-normal levels of the protein.

Gene therapies are very expensive but factor VIII injections cost £100,000 per year.

Haemophilia  effects 2,000 people in the UK.

The study was published in the New England Journal of Medicine.

COMMENT: cost of gene therapies will reduce as they become more common

Mentioned in this article:

Click on resource name for more details.

Barts and The London School of Medicine and Dentistry

Medical and dental school that is part of Queen Mary University of London.

John Pasi

Professor of Haemostasis and Thrombosis at Barts and the London.

The New England Journal of Medicine

Scientific Journal devoted to medical research

Topics mentioned on this page:
Gene Therapy