NHS deadline threatens Batten disease therapy access

A Welsh toddler battles Batten disease as a NHS funding deadline threatens access to a £500,000 therapy.

NHS deadline threatens Batten disease therapy access for toddler

A three-year-old girl from Wales, Katie Hughes, was rushed to hospital after a seizure and later diagnosed with CLN2 Batten disease, a rare and progressive brain disorder with no cure. Doctors initially treated a fever with paracetamol, but the child’s condition worsened and genetic tests confirmed the diagnosis in July. An enzyme therapy exists that can slow the disease, but it costs about £500,000 per year and must be delivered directly into the brain every two weeks via a permanent device. The NHS has a funding window for children already in treatment or who can start by the end of 2025; beyond that, access on the NHS is not guaranteed due to cost and questions about long-term evidence. The family has launched a fundraiser to cover treatment, equipment, and related days out.

This case highlights how medical breakthroughs collide with budget limits. It shows the pressure on public systems to fund expensive, high-stakes therapies for rare diseases while balancing fairness for all patients. The ticking clock compounds the emotional strain on Katie’s family and puts lawmakers under scrutiny about what care should be publicly funded. Beyond the family’s story, the situation raises questions about how the NHS and NICE weigh evidence, cost, and long-term value in a crowded budget.

Highlights

The outcome of this funding debate will matter far beyond a single family.