Breakthrough treatment could help children with rare condition ‘lead happier lives’

Health & Fitness
5 Mar 2026 • 9:25 AM MYT
The Independent
The Independent

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  • An experimental drug, zorevunersen, has shown a dramatic reduction in seizures among young patients with Dravet syndrome during early clinical trials.
  • The collaborative trial, led by University College London (UCL) and Great Ormond Street Hospital (Gosh), involved 81 children aged two to 18 from the UK and the U.S.
  • Zorevunersen works by targeting a faulty gene (SCN1A) to boost the production of a vital protein, aiming to mitigate seizures and associated cognitive and behavioural challenges.
  • Patients who received 70mg of zorevunersen experienced a significant reduction in seizures, ranging from 59 per cent to 91 per cent, over 20 months in extension studies.
  • The treatment has been described as life-changing by participants' families, with researchers hoping it will enable children with Dravet syndrome to lead healthier and happier lives.

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