What changed
A gene therapy for children with Sanfilippo Syndrome Type A has been approved in the United States. FAYUVI, developed by Ultragenyx, is priced at $3.95m per child, or almost £3m, leaving Tate McDaid’s family facing a huge bill as they seek access from Wales.
Why it matters
Tate is turning six this month. His mother, Tammy McDaid, says he is already showing cognitive decline, and that the condition can accelerate after the age of five.
Sanfilippo Syndrome gradually takes away abilities such as walking and eating. FAYUVI could delay that decline and help Tate stay active for longer, but the treatment is not yet available in the UK. Welsh health officials are discussing possible access routes with counterparts in the US, while the cost for international patients remains unclear.
Tate’s family has raised more than £160,000. That is vital progress, but only about 5% of the estimated UK cost. The cruel arithmetic is that a treatment can exist and still be unreachable.
The outlook is uncertain because neither overseas access nor UK funding has been secured. If a pathway is found, Tate’s family would go to the US immediately; if not, time matters because the treatment may be less effective as he gets older.
Comments
No comments yet.