Family faces £3m bill for child's rare dementia treatment

Tammy McDaid, the mother of five‑year‑old Tate McDaid of Swansea, says the only available treatment for his Sanfilippo Syndrome – a rare form of dementia affecting just over 100 children in the UK – now carries a price tag of almost £3 million. The gene‑therapy drug, known as FAYUVI, received its first approval for children in the United States in September. The cost set for each child is £2.95 million, equivalent to US $3.95 million, and it is unclear how families outside the US can fund it.

Tammy told reporters she spent “months praying” for the therapy’s approval before learning of the price, adding “I was excited when the treatment initially got approved but then reality hit hard the next day”. She has contacted the developer Ultragenyx seeking clarity on how non‑American families might cover the expense, noting the company’s response that “We understand what they are charging for American citizens, but they've got health insurance”. She also asked, “What is it for us in the UK?

Not even just the UK, but everyone else in the world looking for this treatment.” Welsh health officials are currently talking with US counterparts to explore possible access routes, while Ultragenyx said “There is not a single estimate we can provide for the total cost of care for an international patient, as those costs would depend on the patient's individual medical needs and the treating institution”.