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Batten disease treatment: Parents win battle ahead of court date | Batten disease treatment: Parents win battle ahead of court date |
(38 minutes later) | |
Families of children with a rare degenerative disease have won their fight for funding for NHS treatment. | |
The four children, from Newcastle and Cheshire, have Batten disease, which is incurable and causes seizures, visual impairment and mobility loss. | The four children, from Newcastle and Cheshire, have Batten disease, which is incurable and causes seizures, visual impairment and mobility loss. |
NHS England confirmed a "deal had been struck" to fund a drug which the families say slows the disease. | |
Mum Gail Rich, of Tyneside, said "every second, minute, hour of campaigning was worth it". | |
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. | 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. | But NHS England said manufacturer Biomarin had agreed a "fairer price" for the drug, which is called cerliponase alfa. |
Seven-year-old Nicole Rich and her sister Jessica, three, from Throckley, Newcastle, currently 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. | Their parents, Gail and Matthew, said since taking it, Nicole had shown no further symptoms and Jessica had not developed any. |
Responding to the decision by NHS England, an emotional Mrs Rich tweeted it was "the day we have been waiting for" and said she and her husband "can't stop crying". | |
Lucy and Mike Carroll, from Poynton, also said the treatment had been beneficial for their eight-year-old, Oliver. | 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. | His six-year-old sister Amelia has not developed symptoms while taking it. |
Earlier this year the families won permission for a judicial review to decide whether the decision not to fund treatment was 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." | |
Children with the condition live until about 10. | Children with the condition live until about 10. |
The families' lawyers said, with the treatment they could live to 60. | The families' lawyers said, with the treatment they could live to 60. |