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Breakthrough gene therapy offers cure for a rare bleeding disorder heamophilia


Key points from article :

A transformational therapy has effectively cured people with the bleeding disorder haemophilia B.

An engineered virus was given instructions to manufacture the missing clotting factor IX.

The virus delivers the blueprints to the liver, which then starts producing the clotting protein.

It was a one-off infusion that takes about an hour to drip.

Nine out of 10 patients given the therapy no longer needed their clotting factor IX injections.

Gene therapy cost, minimum age requirement, and duration of effect remain unknown.

Advances like this could revolutionise the lives of adults with haemophilia in the next few years.

Study led by Pratima Chowdary from University College London, published in the New England Journal of Medicine.

Trial results were positive but cautioned the use until proven safe and effective

Mentioned in this article:

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Pratima Chowdary

Heamatologist and Professor of Haemophilia and Haemostasis at University College London

Royal Free Hospital

Health care provider.

The New England Journal of Medicine

Scientific Journal devoted to medical research.

University College London (UCL)

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