Patients having monthly injections of the drug tofersen reported better mobility and lung function after a year of treatment - with one who was in a wheelchair at the start of the trial now able to walk without sticks.
For reasons that are poorly understood, it causes the nerves that take signals from the brain to the muscles to stop working. That leads to muscle weakness and paralysis that gets worse with time and is ultimately fatal.
New updated results on 108 patients, published in the New England Journal of Medicine, show the biological effect translated into real clinical benefit after a year of continued treatment. Les Wood, 68, from Thorne, South Yorkshire, was diagnosed with MND 10 years ago and first took part in the trial in 2016.
Professor Chris McDermott, from the University of Sheffield Institute for Translational Neuroscience, and another of the researchers, said:"It's having an impact and it's a step-change for people living with this illness, but it's not a cure. The scientists are hopeful that they will be able to use the same drug design techniques to target other genes that can trigger MND, leading to an era of personalised medicine.
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