Intended for healthcare professionals


Gene therapy shows promise for haemophilia

BMJ 2001; 322 doi: (Published 16 June 2001) Cite this as: BMJ 2001;322:1442
  1. Scott Gottlieb
  1. New York

    A new type of gene therapy boosted levels of factor VIII clotting factor in a small number of people with haemophilia, allowing two severely ill patients to go for 10 months without spontaneous bleeding, a new study says.

    These results are far from the proof needed to bring the treatment to market. In particular it is not clear if the effect lasts long …

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