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A five-month-old baby has become the first patient in England treated with a potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy. Arthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May 25. Until two years ago, there were no treatment options available for children with spinal muscular atrophy.
Five-month-old baby becomes first NHS patient to receive drug for genetic condition
Arthur Morgan was diagnosed with spinal muscular atrophy (SMA) earlier this month.
He received the one-off US gene therapy, Zolgensma, at Evelina London Children s Hospital on 25 May.
The treatment has been called the most expensive drug in the world - with a list price of £1.79m per dose.
It was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.
Baby Arthur, who was born six weeks premature in December, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier.