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Newbold girl with rare degenerative brain condition could yet be given a lifeline

Four year old Grace Thompson could get the news her family are so desperate for this week.

A four year old Chesterfield girl, who's being denied access to treatment for a degenerative brain condition known as Batten disease, may be given a lifeline yet. 

The drug Brineura hasn't been approved for use on the NHS by the government organisation NICE, due to it not being cost-effective. 

But a decision's expected in the near future-on a review which could give Grace Thompson access to the treatment- which could extend her life by decades.

Dr Richard Brown is a consultant who works with children diagnosed with Batten disease;

"This is a really exceptional situation, where the treatment is available to doctors now in principle, and yet on the basis of cost alone is being denied.

"I very strongly feel the National Institute of Clinical Excellence should support the use of this treatment."

Meanwhile, Grace's mum Izabela feels there's no time to waste;

"I'd like them to act fast, treat her now and talk about money while she's having the treatment, because Grace hasn't got time.

"Without this treatment, she'll die."

We've tried to contact NICE for an update on their review into Brineura, but they're yet to respond to our request.

However, the company who manufacture the drug-BioMarin-say the annual costs of £500,000 per year reflect the investment required to develop and deliver the highly complex treatment to an exceptionally small patient population.

 

 

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