A BABY given the world’s most expensive drug costing £1.79million four weeks ago has made “huge” progress already, his parents said.
Edward Willis-Hall lifted his head and body from the cushions and held himself for about five minutes thanks to the wonder-drug.
Edward, from Colchester, has a rare form of Spinal Muscular Atrophy (SMA).
He received a dose of Zolgensma last month at Sheffield Children’s Hospital.
Eleven-month-old Edward’s condition means he lacks a protein vital for muscle development.
He has Type One SMA, a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.
Read more >> Baby receives world’s most expensive drug - which costs £1.79million
But, after receiving the drug, Edward is now able to support his head and body.
Edward’s mother, Megan, 30, told the BBC: “Edward lifted his head and body from the cushions and held himself for about five minutes.
“We feel so thankful and blessed. I can’t tell you the difference in him already. He has been doing things he couldn’t do before the treatment as he can now hold his legs up to his belly.
“Edward will always have SMA, but I’m excited for his future as we think this is the best treatment he can have.
“We were told it might take months for any change, but everything about him is a little bit stronger.
“His energy levels and head control most of all.”
Zolgensma was previously unavailable on the NHS and could only be administered privately at an eye-watering cost.
But this changed in June when a five-month-old boy from London became the first to receive a dose when it was approved for use in the UK after months of trials.
After Sheffield Children’s Hospital became one of the four centres approved for the use of Zolgensma for the treatment for Type-1 SMA, Edward is now the fifth patient to receive the drug at the hospital.
Jeff Perring, medical director at the hospital, said: “This treatment will really improve the quality of life for these children and we’re proud to be part of this great advance in treatment using gene therapy.”
About 40 children are born each year with the disease, rarely living past two years old if they do not receive the correct treatment.
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