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A mother from Swansea faces the daunting challenge of raising nearly £3 million for a crucial treatment for her five-year-old son, Tate McDaid, who suffers from a rare and severe form of dementia. Tate was diagnosed with Sanfilippo Syndrome, a condition affecting just over 100 children in the UK, when he was four years old. This progressive disease, often referred to as “childhood Alzheimer’s,” gradually impairs motor skills and cognitive abilities, with symptoms worsening rapidly after age five.
In a significant medical development, the first gene therapy designed to treat Sanfilippo Syndrome in children was approved in the United States in September. While this breakthrough offered a glimmer of hope, Tate’s mother, Tammy, soon faced the harsh reality of the treatment’s cost. The one-time therapy, known as FAYUVI, carries a price tag of $3.95 million (approximately £2.95 million) per patient. Tammy revealed that she had spent months hoping for the treatment’s approval but was devastated upon learning the extraordinarily high cost and the uncertainty surrounding access for families outside the US.
Tammy has reached out to the drug developer Ultragenyx to gain clarity on how international patients without American health insurance might afford the treatment. Ultragenyx indicated that the total costs for overseas patients would vary depending on individual medical needs and their treatment facility, making it difficult to provide a definitive cost estimate. Meanwhile, health authorities in Wales are engaged in talks with US counterparts to explore possible ways to make the therapy available for Tate in the UK.
Sanfilippo Syndrome, also known as Mucopolysaccharidosis type III (MPS III), currently has no cure, though the newly approved gene therapy shows potential to slow down its progression. This delay could help Tate maintain his abilities for longer. Tammy expressed the urgent nature of the situation, emphasizing that time is a critical factor. She explained, “If a millionaire turned around tomorrow and gave us the money, we’d go to the US straight away.” With Tate approaching his sixth birthday and already showing signs of cognitive decline, Tammy fears the window for effective treatment is rapidly closing, leaving her feeling both desperate and terrified about the future
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