"Edward had to have a double hip replacement in October and he's only just getting back on his feet, but in general he is doing so well. "He is learning to swim, he can float on his own, which is really hard for children with SMA because they don't have natural buoyancy. "This summer, he jumped off a boat into the sea and he went on a jet ski. He's a very sweet, loveable little guy."
"We just didn't think that was possible. We didn't know what quality of life he would have,"
"They want to see first-hand what gene therapy has done for him."
Edward is a five-year-old boy from Colchester with spinal muscular atrophy (SMA) who received the one-off gene therapy Zolgensma through the NHS in 2021 at a cost of £1.79m. SMA causes muscle weakness, affects movement and breathing, and about 65 babies are born with SMA in England each year; many do not survive past age two without intervention. Edward progressed from being lethargic as a baby to walking independently, swimming, floating on his own, and taking part in activity such as jumping from a boat and riding a jet ski. He underwent a double hip replacement in October and is recovering. He has started school, made friends, and clinicians have visited to observe the effects of gene therapy.
Read at www.bbc.com
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