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Gortin man living with Huntington’s hopeful after breakthrough

A GORTIN man who has lived with Huntington’s disease for 25 years has welcomed a major medical breakthrough that could transform the lives of families affected by the cruel inherited condition.

Early trial results of a pioneering gene therapy, AMT-130, suggest it can slow the progression of Huntington’s by up to 75 per-cent, potentially giving patients years – even decades – of better quality life.

Huntington’s is often described as a combination of dementia, Parkinson’s Disease and motor neurone disease. It destroys brain cells, shortens lives and casts a shadow across generations.

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For Mickey McNulty, who was diagnosed in his twenties, the news brings cautious optimism.

“When I was first diagnosed, the doctor thought a cure would be found in ten years, but here we are 25 years later,” he said.

“That’s why I’m always cautious. But the idea of slowing symptoms – of being able to enjoy my retirement – would be absolutely incredible.”

Now aged 50 and still working, Mickey said the prospect of stabilising his health would be ‘life-changing’.

The new therapy is delivered through intricate brain surgery lasting up to 18 hours. Once in place, AMT-130 works to protect and repair neurons – the brain cells damaged by the disease.

Patients in the trial not only experienced slower progression, but also showed measurable improvements in markers linked to brain cell health.

Cath Stanley, BEM, chief executive of the Huntington’s Disease Association, described the findings as ‘remarkable’ and ‘a significant breakthrough’.

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Biopharmaceutical company UniQure plans to seek regulatory approval in the US next year before applying in Europe and the UK.

The final stage will be a review by NICE, which will decide whether the treatment can be offered on the NHS.

The Omagh Huntington’s Support Group, which meets regularly, also welcomed the news.

“This is great news for people with Huntington’s disease and will fill people with optimism about the future,” a spokesperson said.

The group will host its Family Conference at the Silverbirch Hotel on Saturday, October 18.

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