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Haemophilia – Trial results are very impressive

British doctors say they have achieved “mind-blowing” results in an attempt to rid people of haemophilia A.

Patients are born with a genetic defect that means they do not produce a protein needed to stop bleeding.

Thirteen patients given the gene therapy at Barts Health NHS Trust are now off treatment with 11 producing near-normal levels of the protein.

Jake Omer, 29 from Billericay, Essex, was on the trial and says he feels like he has a new body.

Like 2,000 other people in the UK, his body could not make clotting factor VIII.

A minor injury used to cause severe bleeding. He remembers losing two front teeth as a child and bleeding for days afterwards.

Even the impact of walking would lead to bleeding in his joints and eventually cause arthritis. Jake has needed at least three injections of factor VIII a week for most of his life.

But in February 2016, he had a single infusion of gene therapy.  The first time he knew it had worked was four months after the therapy when he dropped a gym weight and bashed his elbow. He started to panic, but after icing the injury that evening, everything was normal the next day.

The therapy is a genetically engineered virus. It contains the instructions for factor VIII that people are born without. The virus is used like a postman to deliver the genetic instructions to the liver, which then starts producing factor VIII.

Gene therapies are likely to be spectacularly expensive. However, the current cost of regular factor VIII injections is about £100,000 a patient per year for life.

This is Massive news and a leap forward in the right direction.

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