Parents of children with Batten's disease are "terrified" by funding decision



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Amelia and Ollie Carroll

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Amelia and Ollie Carroll, now eight and six years old, from Poynton, have Batten's disease

The parents of a brother and a sister with a rare and hereditary disease said that they were "devastated" and that their treatment would not be funded by the NHS.

Ollie and Amelia Carroll, aged eight and six, from Poynton, have Batten's disease, which is currently incurable.

Their parents campaigned for a drug that could slow their progression and be recognized by the NHS in the long run.

But the use of the NHS will no longer be recommended for reasons of "concern about its long-term effectiveness".

The National Institute of Health and Care Excellence (NICE) said that the decision not to recommend the drug – cerliponase alfa – came at the end of "year-long negotiations" "between the company that produced it and NHS England.

& # 39; They will die & # 39;

He said that the company was not able to "fix the price of treatment" at a level that would have solved the problems highlighted in its badessment by NICE.

In a Facebook post, parents Lucy and Mike said, "As parents, we are completely devastated by this decision."

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Pennsylvania

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Ollie meets Prince Harry in 2016

The statement added: "We do not know what this decision means for Ollie and Amelia, but it's terrifying.

"If the treatment is interrupted, Ollie and Amelia will deteriorate quickly and eventually they will die."

The parents said they took legal advice and aim to continue to fight for their right to use the drug.

& # 39; Short term benefits & # 39;

In its previous draft guidelines, released last year, the independent NICE committee had agreed that, although alpha cerliponase is not a cure, it was "an important development". to treat the disease ".

He also said the well had shown "substantial short-term benefits by slowing down the rate at which it is progressing".

Symptoms in children with the disease begin around the age of two and can then evolve rapidly. These included: –

  • seizures
  • decline of speech
  • loss of mobility
  • involuntary muscle spasms
  • progressive dementia
  • visual impairment leading to blindness

The average life expectancy of a child with this disease is 10 years. It is estimated that about 30 to 50 children live in the UK with Batten's disease.

The NICE Guidance Project is now in consultation.

The pharmaceutical company, health professionals and patient / caregiver organizations all have the opportunity to appeal recommendations.

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