There’s further hope for a 12-year-old and her family battling for her to access a potentially life-saving drug after her condition was brought up in Parliament.
Rae White, of Gedney Hill, hopes the drug Spinraza will stop the type two Spinal Muscular Atrophy (SMA) she’s had since birth from further limiting her movements.
And after numerous delays there’s hope she will be among those who will be able to access it within three months as mentioned in a House of Commons debate held after South Holland and the Deepings MP Sir John Hayes took up the family’s fight.
Holland and the Deepings.
At the debate Seema Kennedy, the Parliamentary Under-Secretary for Health and Care, said: “I am really pleased that only yesterday, NICE (National Institute of Health and Care Excellence) published its final guidance, meaning that this debate is very timely.
“The guidance recommends the use of Spinraza for many patients with SMA through a managed access agreement, which has been negotiated between NICE, NHS England and the manufacturer, Biogen.
“This makes Spinraza available to the NHS at a discounted price.”
She continued: “Following yesterday’s announcement, eligible patients with types two and three will begin treatment as soon as possible within the next three months.”
Sir John argued: “The real issue here is whether Spinraza would benefit every single sufferer. If it would, it should be universally available.”
Rae and Tanya are now waiting to speak to their consultant to see if Rae is eligible and can start treatment in the hope it allows her to at least continue the use of her arms which the condition has started to have an effect on.
Tanya said: “We’re still in the dark.
“We’ve got to speak to the consultant. I know some people have started getting their referral dates, but it’s all a little bit up in the air.
“It’s definitely a positive and it looks like things are going in the right direction.
“Sir John Hayes was fantastic and spoke brilliantly on the subject.
“He came over and spoke to us like we weren’t just robot and I feel like hearing our story affected him.”
Sir John added: “It’s a really strong example of a conversation driving something that MPs can take and push up the agenda.”
A further boost to the Whites is the announcement of a new drug being tested called Risdiplam.
Rather than boost missing proteins via an injection in to the spine as Spinraza is, this is delivered orally.
“It’s supposed to be much less expensive and would be safer to administer,” she said. “But it will have to go through all the processes Spinraza has so we will see.”
