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Caroline Scott

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A drug, Spinraza, which helps delay the progress of the neuromuscular disorder spinal muscular dystrophy (SMA)

is approved for use in the US and Europe, but flaws in the way medicines are approved in the UK, means children here are missing out.

Read more here

Consultant endocrinologist, Professor Peter Hindmarsh
“I’m still me, I’m still here..”
Caroline Scott 2025
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