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'Game-changer' drug saves life of Scots baby suffering with incurable muscle-waste condition

Daily Record logoDaily Record 14/06/2021 Vivienne Aitken

A tiny baby’s life has been saved by a miracle “game-changing” drug.

Isabella Winfield was diagnosed with Type 1 spinal muscular atrophy at just 11 days old.

Ninety per cent of tots with the incurable muscle-wasting condition do not survive past their first birthday.

But six-week-old Isabella is thriving after becoming one of the first babies in the UK to be treated with Zolgensma, a drug now available on the NHS after a deal was struck with its manufacturer Novartis.

Her parents Margaret and Richard thanked the team caring for her at the Royal Hospital for Children in Glasgow and for giving her the chance of a normal, healthy life.

Margaret, from Archiestown, Moray said: “I was aware of Zolgensma but thought it was still in trials.

"As soon as they said it was available to us, we just thought yes, go for it. We had no hesitation. If it’s available for her, it’s her best hope.”

Zolgensma reduces the need for artificial ventilation in babies with SMA and it can help babies sit unaided for at least 30 seconds after 18 months, a milestone not achieved in untreated babies with severe forms of the disease.

Isabella’s neurologist Dr Iain Horrocks said: “Zolgensma is a game changer. Isabella was the ideal candidate.

“We only treated her about three weeks ago so it is too early to talk about the future, but we are, of course, optimistic.”

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