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PostPosted: 14 May 2019 22:37 
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A hand surgeon who faced losing the ability to perform operations said a new drug has saved his career.

Carlos Heras-Palou, an orthopaedic surgeon at Royal Derby Hospital, was diagnosed with transthyretin-mediated amyloidosis (hATTR amyloidosis). The hereditary condition affects limb movement and other functions, and can be fatal in some cases. hATTR amyloidosis would have stopped him from performing operations

The 53-year-old took part in a trial for patisiran, which blocks the activity of a gene in the liver. Having been diagnosed early after suffering "pins and needles and intermittent numbness", he said the "ground-breaking" treatment has been "like the proverbial silver bullet".

"The treatment has saved my career, and my life," he said. "I'm left with some numbness in my feet and weakness, and I still can't run, but I can walk. I'm functioning very well. I've been given hope and I'm seeing life in a different way."

The trial involving 148 patients being treated with patisiran and 77 with a dummy placebo was published in the New England Journal of Medicine. It concluded that the disease progression was "halted or reversed" in patients who received the active drug.

Patisiran has now been licensed by European and UK regulators. However the drug is very expensive at the moment and is currently priced at about £308,618 ($400,000) per patient per year. As a trial participant, Mr Heras-Palou has been allowed to continue receiving patisiran, About 100 people in the UK are believed to suffer from hATTR amyloidosis.

It is now being reviewed by the National Institute for Health and Care Excellence (Nice), which approves new NHS treatments in England, and final guidance on the drug is expected sometime this year.

Professor Philip Hawkins, director of the National Amyloidosis Centre and one of the scientists involved in the trial, said the results were "wonderfully encouraging".

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