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Duchenne muscular dystrophy: ‘Breakthrough’ drug for severe muscle wasting condition set for NHS rollout in England

The Independent·Storm Newton·25 days ago
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Hundreds of children across England are set to benefit from a new drug which has been approved for rollout on the NHS to treat a severe muscle-wasting condition. Givinostat is expected to enable eligible patients with Duchenne muscular dystrophy (DMD) to maintain their mobility for longer. The National Institute for Health and Care Excellence (Nice) confirmed the drug's availability after its manufacturer reached a commercial agreement with NHS England. This decision marks a significant step for families affected by the rare genetic disorder. While campaigners welcomed the long-awaited approval, they highlighted the "agonising" two-year process, during which many families were left without access to the drug as their child's condition continued to deteriorate. Duchenne muscular dystrophy is caused by a lack of the protein dystrophin, leading to the breakdown of muscle fibres. Primarily affecting boys, an estimated 2,005 individuals live with the condition in the UK.…

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