New gene therapy for children's dementia costs nearly £3 million

A gene therapy for Sanfilippo Syndrome, a rare form of dementia affecting just over 100 children in the UK, was approved in the US in September, BBC Health reported. The treatment is designed for children and represents a significant development in managing this condition.

The therapy, developed by Ultragenyx and marketed as FAYUVI, is priced at approximately $3.95 million (around £2.95 million) per child in the US. This cost has raised concerns about accessibility for families outside the US, especially those without health insurance.

A mother of a five-year-old boy diagnosed with Sanfilippo Syndrome expressed both hope and concern after the treatment's approval. While initially excited, she realized the financial burden the therapy would impose, as funding options for UK families remain unclear.

Health officials in Wales are currently in discussions with US counterparts to explore potential pathways for access to the treatment for children like her son. Ultragenyx acknowledged the pricing for American citizens with insurance but has not provided estimates for families outside the US.

The situation highlights challenges in accessing cutting-edge treatments internationally, especially for rare diseases where therapies may be costly and funding mechanisms vary by country.