A mum who says she’s watching her 11-year-old daughter slowly waste away has been left distraught over the delay of a potential treatment.
Rae White, who turns 12 next week, was born with type two Spinal Muscular Atrophy (SMA) a condition that slowly stops her body functioning.
She has always been wheelchair bound and is now also starting to lose the ability to use her arms.
Hope came two years ago when a drug called Spinraza was discovered that has been shown to stop the deterioration of the body.
Rae, of Gedney Hill, is quick to tell you that it’s available in 23 European countries, including Scotland.
But because just one private company called Biogen has the expertise it is expensive. Rae’s mum Tanya White says it would cost the NHS £200,000 a year per child.
In May the National Institute of Health and Care Excellence announced it had been recommended for all SMA patients to be approved, something Tanya broke down in tears of joy when she heard.
However its roll out has been delayed and Tanya has been told Rae may not now be eligible for the treatment.
Rae is in the top three sets for all subjects at Universtiy Academy Holbeach. Her favourite subject is maths and she hopes to do something involving that as a career, such as banking.
She describes her condition as “hell”.
“I can’t do anything,” she said. “I have no independence whatsoever.
“You’re constantly having to ask anyone around you to do things. I can’t even turn a light switch on or scratch my head.
“A few years ago I was able to pick up a cup, but I can only use a very small one now.”
Tanya added: “It’s an awful condition. Rae was a proper fidget when she was little.
“She could turn herself in bed, crawl around and play. Now once she’s laid down she might as well be paralysed as she can’t even lift her own head.
“As a parent one of your main jobs is to protect your children and to watch your child slowly waste away is heartbreaking.
“There’s nothing you can do, you’re powerless.
“We’re watching other kids get treatment and it is amazing to see the strength they’re gaining back, yet we’re watching our loved one deteriorate knowing treatment is in arm’s reach but not quite there.”
Rae is also currently waiting for spinal treatment and on both dislocated hips caused due to her condition.
She said of the potential new treatment: “I’ve always seen it as if I don’t get my hopes up then I won’t be disappointed as I know eventually they will say I can’t have it.
“If I get my hopes up I’m going to be destroyed if I can’t have it.”
Tanya continued: “As an adult when you’re caring for someone all you can do is live in hope.
“When Rae was diagnosed we were told ‘take your child home and enjoy them because there is no treatment and no cure’.
“Two years ago it was announced there is a treatment, something we’d never thought we’d see.
“Everyone was so elated, I can’t tell you how amazing that felt.
“We battled for two years for them to announce it would be approved with no restrictions. Two weeks later they say there will be restrictions.”
Rae thinks three quarters of all SMA patients could be excluded though the family don’t know if she is one of those.
“It’s like chucking kids in water and saying who gets a life jacket. That one child is more important than another child,” Tanya said. “To me that’s wicked. I’m not going to take a life jacket off somebody else’s child to give to mine.
“As a parent I will keep her afloat as best as I can.”
Rae added: “I’m just going to try and enjoy my life.”
