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Hundreds more children to get ‘life-changing’ SMA treatments on NHS following Jesy Nelson campaign

The Independent·Ella Pickover·19 days ago
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Hundreds more children suffering from rare muscle-wasting condition spinal muscular atrophy (SMA) are set to gain access to "life-changing" treatments following their approval for widespread use across the NHS . One mother, whose son benefited from an early access scheme for one of these therapies, attested that it had "fundamentally changed the course of his life". The injectable drug nurinersen, which marks the first disease-modifying therapy for SMA, alongside the oral treatment risdiplam, were previously available through a special access programme while further evidence was gathered. Now, the National Institute for Health and Care Excellence (Nice) has given its endorsement for the routine and widespread application of these treatments within the NHS in England. In its final draft guidance, Nice highlighted that the drugs have the potential to improve survival rates, slow the progression of the disease, and help individuals maintain their independence.…

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