Toddler Batten disease funding window

Three-year-old Katie Hughes faces a rare Batten disease and a costly treatment with an NHS funding deadline.

Toddler fights Batten disease as NHS funding window ends

Katie Hughes, aged three, was rushed to hospital after a seizure that doctors initially blamed on a fever. Genetic testing later confirmed CLN2 Batten disease, a rare and deadly brain disorder that will gradually steal her ability to walk, talk and see. Doctors warned she may not live past age eight to twelve.

There is no cure for the illness, but a £500,000 a year enzyme therapy can slow its progress. The treatment must be delivered directly into the brain every two weeks via a permanent device. The NHS has a special deal for children who have started treatment or who can start by the end of 2025, but new patients after that may not receive NHS funding because there is not enough long-term evidence and the budget is tight. Katie's family has launched a fundraising push and is considering private or overseas options.

This case shows how rare disease care sits at the intersection of medicine, money and policy. It highlights a painful truth: the life of a child can hinge on whether a drug exists and whether a health system can afford it. The deadline puts families under pressure while policymakers weigh evidence and budgets, a combination that tests both compassion and sound budgeting. The broader question is how to balance access, equity and scientific proof in high-cost treatments.

Highlights

Policy makers face tough choices as rare diseases press NHS resources