What is CLN2 Batten Disease?

CLN2 Batten Disease is a rare form of childhood dementia that progressively impacts a child's ability to communicate, walk, and eat independently.

What treatment is available for CLN2 Batten Disease?

A treatment involving regular brain infusions is available, which aims to slow the disease's progression and extend the child's life, though it is not a cure.

Why is there a debate about drug access for this rare disease?

The drug's high cost, exceeding £500,000 per year per child, and questions about its long-term effectiveness have led to reviews regarding its continued access via the NHS.

Home / Health / Girl's Rare Dementia Battle: A Race Against Time

Girl's Rare Dementia Battle: A Race Against Time

29 Jan

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Summary

Childhood dementia diagnosed at three, impacting communication and mobility.
Treatment offers extended life but is not a cure, buying crucial years.
Family advocates for government to secure future drug access.

Girl's Rare Dementia Battle: A Race Against Time

Holly Nayler was diagnosed with CLN2 Batten Disease, a rare form of childhood dementia, shortly before her third birthday. This progressive condition will lead to a loss of communication, mobility, and the ability to eat independently. Without treatment, life expectancy for children with this disease is typically between five and eight years old.

The family has opted for a treatment involving regular brain infusions, which, while not a cure, aims to slow the disease's progression and extend Holly's life into her late teens. This treatment is administered via the NHS, following an access agreement that is currently under review.

Despite the significant costs associated with the drug, estimated at over £500,000 per year per child, James Nayler, Holly's father, is urging the government to ensure continued access. He highlights that the drug has significantly improved the quality of life for many children, even though Holly's seizures have worsened.