Five-Month-Old Baby Becomes First NHS Patient to Receive Zolgensma Drug for Genetic Condition

Source: SKY News


A five-month-old baby with a spinal disorder has become the first NHS patient in England to be treated with a potentially life-saving drug that can prolong life. 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.

Until two years ago, no treatment options were available for children with the condition.

Babies born with Type 1 SMA – the most common form of the condition – experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years. But studies have found that a single Zolgensma treatment can help babies with the condition to sit, crawl and walk, and also prevented them from having to be put on a ventilator.


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