Published: 00:01, 24 August 2018
The mother of a teenager with a rare muscle disease is battling to have a life-extending drug approved on the NHS.
Abigail Bolt lives with Spinal Muscular Atrophy (SMA), an incurable muscle weakening condition.
Although there is no cure, the 14-year-old's mum Mel is fighting to have a miracle treatment named nusinersen – also called Spinraza – made available on the NHS.
Ms Bolt, 32, said: “This is the only drug out there for the condition but it’s not available in this country.
“A number of other countries, including America, have trialled it and have seen lots of children progress with it and get stronger but the NHS won’t pay for it.”
She added: “SMA is a muscle wasting condition. The longer Abbie doesn’t have the treatment, she’s getting weaker and weaker.
"She’s deteriorated a lot in the past year.
“By her not getting the drug, I am going to lose her a lot quicker than we should.”
The Westlands School pupil was diagnosed with SMA aged two and is now a full-time wheelchair user.
“Abbie can’t stand, walk, she can’t lift her hands up to brush her hair – all the normal things a 14-year-old girl wants to do,” Ms Bolt said.
"It's just not fair... I've already seen such a change in Abbie since she heard the news - it's heartbreaking" - Mel Bolt
The mother-of-two, from Minster, said the drug Abbie needs is available in 22 countries including Canada, Japan and Scotland.
But the National Institute for Health and Care Excellence (NICE) said, in its draft guidance published last week, it could not recommend nusinersen be used by the NHS to treat the disease.
It said although clinical trial evidence showed the drug had “improved a range of outcomes that are important to patients”, it felt the long-term benefits were “highly uncertain”.
It also said the cost was “too high for it to be considered a cost-effective use of NHS resources”.
Ms Bolt said: “It’s made me feel worried, that Abbie is going to get worse, and sad – it’s a case of there is this drug out there but she’s not getting the benefits of it.
“To watch your child deteriorate in front of you is so awful and there’s nothing I can do.
“That’s why I’m trying to raise awareness about the condition and get NICE to realise there is a bigger picture to this.”
Ms Bolt has teamed up with TreatSMA, a community of people affected by SMA, who are fighting for the treatment.
She said: “We have two months to try and persuade NICE to change its guidelines. It’s just not fair. I’ve already seen such a change in Abbie since she heard the news – it’s heartbreaking.”