Therfield mum says fight is on for drug to help young Courtney
PUBLISHED: 08:24 30 August 2018
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The mum of a nine-year-old girl from Therfield with a genetic condition that means she can’t stand or take part in games with her friends has said she is devastated to learn that a drug that could help has not been recommended for approval on the NHS.
Julie Copeland has been Courtney’s full-time carer since she was diagnosed at 18 months with spinal muscular atrophy type 2.
She told the Crow: “When Courtney was about 12 months old, I realised she wasn’t weight bearing on her legs, and I took her to the doctors who said she was lazy.
“Eventually someone took us seriously, and she was sent to Great Ormond Street where we got that diagnosis at 18 months. I was devastated.
“From then on, our lives spiralled out of control, with endless appointments – it was all about what we can do and what we can’t do.
“It was easier in those days, because I could lift Courtney – now she has to be hoisted. She used to be in a mobile stander and she lost the ability to do that. Her legs now stay in the sitting position.
“Courtney is such a happy girl – it doesn’t bother her that she’s in a wheelchair – but there are some things she gets upset about, like when the other kids were playing with water guns during the hot weather and she couldn’t, and when she drops things she can’t pick them up.
“We are very close, it’s just the two of us. Most of the time I’m good, but sometimes I get tired. I have to turn her in the night five or six times, but we manage.
“I want her to have the best life possible, so we need this medication.”
To Julie’s dismay, the National Institute for Health and Care Excellence – NICE for short – announced two weeks ago it would not recommend a drug, called spinraza or nusinersen, for NHS England – though it is available in Scotland.
The NICE committee noted in its draft guidance that: “The evidence showed a substantial benefit for nusinersen, but felt there were significant uncertainties, particularly around its long-term benefits. The cost of nusinersen is also extremely high.”
Julie, who believes the drug could make a big difference, said: “I’m sitting here like all the other mothers watching my child get weaker and weaker and knowing there’s a drug out there that can make them stronger and not make the symptoms any worse.
“I just think its awful that they are putting a price on children’s lives.”
Courtney is due to start at Roysia Middle School in September and mum Julie hopes the approval will still go ahead so she can live life to the full.
She said: “Courtney is very bright so her condition doesn’t affect her at all that way. It’s just she has no movement in her arms or legs.
“She’s got a fantastic sense of humour, she’s very cheeky, she’s very loveable.
“If we don’t get the drug, she’ll get weaker and weaker. As her body grows, the muscles won’t grow with her, so she’ll lose what abilities she has now. We need that drug, and the fight is on.”