Say hi to Eli – and if you know the name of Harry Potter’s owl and how to build the perfect Lego tower then you’ll get along just fine. Eli’s mum, Emily said: “Anything building related seems to be...
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Edward’s story: “the world is his oyster” after gene therapy treatment
11 August 2021
The mum of eleven month old Edward says “he has been given a future” after receiving gene-therapy for Spinal Muscular Atrophy. Baby Edward came to Sheffield Children’s to receive the potentially life-saving drug Zolgensma, which can prevent paralysis and...
First patient in Yorkshire given potentially life-saving gene-therapy drug at Sheffield Children’s
07 July 2021
A two month old baby is the first patient in Yorkshire to receive a potentially life-saving drug that can prevent paralysis and prolong the lives of children with Spinal Muscular Atrophy (SMA). Baby Markuss received the drug Zolgensma, which...
Elijah’s story: Travelling to Sheffield Children’s for SMA treatment
04 May 2021
Meet five year old Elijah, he lives in Leicestershire and travels up to Sheffield Children’s every 16 weeks for treatment. In 2019, Elijah was diagnosed with Spinal Muscular Atrophy (SMA) Type 2 after a genetics investigation, following an earlier...
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