Drug breakthrough for children with severe form of epilepsy
Drug breakthrough for children with severe form of epilepsy 8 hours ago Michelle RobertsDigital health editor A new drug is transforming the lives of children born with a severe form of epilepsy, say doctors and families.
Without effective treatment, the outlook can be devastating, with uncontrolled fits putting children at high risk for injury and death.
His mum Lauren told BBC News that the drug has been a game-changer for eight-year-old Freddie.
"We now have a life we didn't ever think was possible and, most importantly, it's a life that Freddie can enjoy," she said.
"Before treatment, life was difficult.
Since, he's climbed mountains, we can go out walking with the dogs, walk around the lakes. And he's even been skiing. " The early trial results, published in the New England Journal of Medicine, show the experimental treatment can be given safely to adolescents and young children, from the age of two onwards.
It means only half of the normal amount of sodium channels are produced in some neurons.
Prof Helen Cross, one of the lead researchers from University College London's Institute of Child Health and Great Ormond Street Hospital, says the results have been truly promising.
"And even perhaps, if we get the treatment right, get near normal living in the longer term. "
Many of the patients, including Freddie, are continuing on the medication for ongoing research.
Dravet Syndrome UK chair of trustees Galia Wilson said: "We regularly see the devastating impact that this condition has on the lives of families. That's why we're so thrilled about these latest results from the initial zorevunersen clinical trials
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