Batten disease treatment: Parents win battle ahead of court date

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Nicole Rich has had treatment at Great Ormond Street Hospital as part of a drugs trial

Families of children with a rare degenerative disease have won their fight for funding for treatment.

The four children, from Newcastle and Cheshire, have Batten disease, which is incurable and causes seizures, visual impairment and mobility loss.

Their parents had been refused funding for an expensive drug which they say slows the disease down.

But NHS England has now confirmed that a “deal had been struck” to fund the “life-changing” treatment.

The National Institute for Health and Care Excellence (NICE) had previously said it could not be certain it was value for money. The treatment costs £500,000 per child per year.

But NHS England said manufacturer Biomarin had agreed a “fairer price” for the drug, which is called cerliponase alfa.

‘Flexible and realistic’

Earlier this year the families won permission for a judicial review to decide whether the decision not to fund treatment is legal. The hearing was scheduled for next month.

A NHS statement said: “This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments.

“Coming after extended negotiations, the new deal reached today is a reminder that in order to succeed companies must be flexible and realistic.”

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The parents of Amelia and Ollie Carroll say the treatment has been beneficial

Seven-year-old Nicole Rich and her sister Jessica, three, from Throckley, receive the drug through a scheme funded by an America pharmaceutical firm.

Their parents, Gail and Matthew, said since taking it, Nicole had shown no further symptoms and Jessica had not developed any.

Mrs Rich said the treatment had “given us real hope for the future”.

Lucy and Mike Carroll, from Poynton, also said the treatment had been beneficial for their eight-year-old, Oliver.

His six-year-old sister Amelia has not developed symptoms while taking it.

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Jessica travelled to Hamburg for treatment until last year when she was allowed join her sister in London

Children with the condition live until about 10.

The families’ lawyers said, with the treatment they could live to 60.