@bozzalad | ||
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 |
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@bozzalad | 14 December 17 | |
Jake told the BBC: I feel like a new person now - I feel like a well-oiled robot. I feel I can do a lot more. I feel my body allows me to do more. I don't think I would have been able to walk 500m without my joints flaring up, whereas now I think sort of two, three, four-mile walk - I could quite easily achieve that. 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. |
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@bozzalad | 14 December 17 | |
Great news for many.
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@bozzalad | 14 December 17 | |
The therapy is a genetically engineered virus. It contains the instructions for factor VIII that Jake was born without. The virus is used like a postman to deliver the genetic instructions to the liver, which then starts producing factor VIII. In the first trials, low doses of gene therapy had no effect. Of the 13 patients given higher doses, all are off their haemophilia medication a year on and 11 are producing near-normal levels of factor VIII. Prof John Pasi, who led the trials at Barts and Queen Mary University of London, said: This is huge. It's ground-breaking because the option to think about normalising levels in patients with severe haemophilia is absolutely mind-blowing. To offer people the potential of a normal life when they've had to inject themselves with factor VIII every other day to prevent bleeding is transformational. An analysis of the first nine patients on the trial was published in the New England Journal of Medicine. |
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@birdy | 14 December 17 | |
I hope it works. This is really rough.
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@shadow27 | 14 December 17 | |
One of my favourite musicians was born with it, Baterz.. He died in 2002. He wrote a song about it, trying to find the lyrics, ooh.
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@sisfreak2017 | 14 December 17 | |
Are that's great news , its a very cruel illness, a friend at school had that , just a scratch used to look like someone had run a knife across the skin ,he used to regular have to visit schools nurse for injections of I think something called factor8 , lost touch after school hope he's still around.
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@shadow27 | 14 December 17 | |
Baterz was known for including offbeat subject matter in his lyrics: whether haemophilia, diabetes, car accident victims, amputees, drowned television personalities or even arachnid mothers, along with many other diseases and mishaps of human condition, probable or otherwise, Baterz was said to have even made pessimism funny - a particularly impressive feat considering that in 1984 he was diagnosed with HIV passed on during a routine blood transfusion a year earlier. Despite this debilitating virus, Baterz remained tireless both as a constant touring solo artist (releasing three singles and two albums: 'Baterz Out Of Hell' and 'Live And Well') and occasionally reuniting with The Bedridden. March 2002 saw the release of a new Bedridden single, Inland Sea, which was to herald a fourth album. Yet as fate would have it, around the same time lesions were discovered in his brain, hospitalising him for a couple of months and eventually stripping him of all coordination. Poor fella. I met him once, used to hear him on the radio all the time. |
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@shadow27 | 14 December 17 | |
https://youtu.be/WrTF2bhwYhc That's the Haemophiliac song.. Can someone else make that a link for me? Ty. |
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@ogdenz | 14 December 17 | |
Fantastic news.
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@birdy | 14 December 17 | |
I was made aware of how brutal this sh*t is when I read April Fool's Day by Bryce Courtenay. Pretty heartbreaking. I never knew of Baterz I don't think. He seems cool though.
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