Family of UK boy with rare dementia faces £3m gene therapy bill

A Swansea mother says her young son lives with a rare illness that affects the brain, and the treatment that could slow it costs almost £3m. According to BBC News, Tate McDaid received a diagnosis of Sanfilippo Syndrome at the age of four. The BBC reports that only a little over 100 children in the UK are thought to have it.

In September, the US approved the first gene therapy for children with Sanfilippo Syndrome. The drug, called FAYUVI, is made by Ultragenyx. Its one-time US price is $3.95 million, while the cost for UK families has been set at £2.95 million per child. It is not yet clear how families living outside the United States could pay for it.

A mother’s search for funding

Tate’s mother, Tammy, said she prayed for months for the approval. She told the BBC she felt excited when the news came, but the price changed that feeling the next day. She said she found the figure hard to accept.

Tammy contacted Ultragenyx to ask how families outside the US, who usually lack American health insurance, could cover the cost. The company said it could not offer one overall estimate for an international patient’s care. It explained that such costs would depend on each patient’s medical needs and the hospital treating them.

Tammy has raised more than £160,000 so far. The BBC reports that this equals only about 5% of the estimated total. She said the family needs millions, not hundreds of thousands, and she hopes the drugmaker might later offer a lower price.

Why time matters

Sanfilippo Syndrome Type A, also known as Mucopolysaccharidosis type III, has no cure. It is sometimes described as childhood dementia because it damages the brain over time. It gradually takes away abilities such as walking and eating.

The BBC reports that the condition can advance faster once a child passes the age of five. The new drug may delay its onset and help children stay active for longer. Tammy said Tate already shows signs of cognitive decline, and she believes the treatment will work less well as he gets older.

Tammy described her son as a happy child who is largely unaware of his illness. He has never spoken, but his mobility matters greatly to him, she said. That is why she is fighting to secure the treatment for him.

Official response and next steps

Health officials in Wales are in talks with US contacts to find out what access routes might exist for Tate. Doctors at Noah’s Ark Children’s Hospital in Cardiff are raising the matter with American officials on the family’s behalf, the BBC said.

Ultragenyx said FDA approval is an important first step in its plan to bring the treatment to families worldwide. It said it understands why some families think about travelling to the US for gene therapy. The company advised families to speak with their child’s doctor about possible access routes before assuming the US is their only option.

The Society for Mucopolysaccharide Diseases, a charity that supports children with Sanfilippo Syndrome, told the BBC that about 140 children in the UK have the condition. This figure differs from the BBC’s own earlier figure of just over 100, so the exact number remains unclear. For now, the family’s main concern is whether funding and access can be arranged before the disease advances further.

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Sources

• BBC News: There's finally a treatment for my five-year-old's dementia – but it'll cost me £3m

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