Wolverhampton mother and daughter join protest march
An 11-year-old girl with a rare muscle wasting disease was one of hundreds taking part in a protest march today as they continue to wait for a new wonder drug to be approved in England.
Heidi Prescott-Booth, from Wolverhampton, has spinal muscular atrophy (SMA), a genetic condition that causes progressive muscle weakness and loss of movement.
The National Institute of Health and Care Excellence (NICE) is today meeting to decide whether to recommend funding for the only-known treatment – nusinersen, also called Spinraza.
Campaign group TreatSMA had organised a protest march outside the NICE offices in Manchester in a bid to raise awareness of the plight of patients.
Heidi and her mother Katie had chosen to attend the march to ensure the schoolgirl's voice was heard.
There are different types of SMA ranging from very severe type 1 through to type 4 – Heidi has type 3 – and one in 40 people carry the gene.
During the past two years her condition has worsened to the point where she can only walk a few steps and is losing the strength in her arms.
Katie, aged 33, believes the treatment would stop her from deteriorating further and would prevent her from losing her independence.
"Heidi just wants to get the treatment. She can't understand why she can't have the treatment when there is one available," she says.
NICE had previously ruled out recommending funding for Spinraza saying it wasn't 'a cost-effective use of NHS resources'.
But since then further meetings have been taking place with Biogen which makes the drug and a revised submission for the NICE appraisal committee was being considered today.
The situation has been made even more agonising for families in England as it was recently announced that Spinraza would be made available on the NHS in Scotland for children and adults with SMA Types 2 and 3 from April.
It follows approval through a new pathway assessment process for the rarest conditions, which was set up in October by the Scottish Government.
Katie said they were trying not to get their hopes up that NICE may give the new drug the go-ahead.
"We would like to think they would follow suit. We've had to wait a long time as the decision was originally going to be in November.
"I hope they see that it's about more than just money, it's about humans.
"There are a lot of children that want and need treatment and they're not just going to go away," she added.
The pair have been supported by Wolverhampton South West MP Eleanor Smith who raised their plight in the House of Common and met them at the weekend.
“Heidi does not have the time for this decision to be delayed or for NICE to rule Spinraza is too expensive.
"As long as I’m MP this is not going away. We’ve got to look at our children as humans not numbers. I will not let this go until it’s the same in England as it is in Scotland.”
Also due to attend the march was Hannah McKeown and her daughter Tiegan, from Gornal,
