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Huntington's disease treated for first time

One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.

Levels of neurofilaments in spinal fluid – a clear sign of brain cells dying – should have increased by a third if the disease continued to progress, but was actually lower than at the start of the trial. Dr Walid Abi-Saab, the chief medical officer at uniQure, said he was "incredibly excited" about what the results mean for families, and added that the treatment had "the potential to fundamentally transform" Huntington's disease. She is already working with a group of young people who know they have the gene, but don't yet have symptoms – known as stage zero Huntington's – and is aiming to do the first prevention trial to see if the disease can be significantly delayed or even stopped completely.

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