A Swansea mother is urgently raising £1.5 million to secure a breakthrough gene therapy for her five-year-old son, who has been diagnosed with a rare form of childhood dementia known as Sanfilippo syndrome.
Race Against Time for Tate
Tammy McDaid's son, Tate, was diagnosed with Sanfilippo syndrome type A in September. The condition, also called childhood Alzheimer's, is a neurodegenerative metabolism disorder that gradually causes children to lose all learned skills, including walking, eating, and moving independently. According to the Society for Mucopolysaccharide Diseases, only 140 children in the UK live with this condition.
Current NHS treatment primarily focuses on pain management rather than preventing cognitive decline. Tammy is now fundraising for a gene therapy treatment that, if approved by the US Food and Drug Administration (FDA), could be the first ever available treatment for this rare paediatric disease. The treatment does not reverse symptoms but is designed to prevent further cognitive decline. Fortunately, Tate has not yet shown signs of cognitive decline, but he will without treatment.
Cost and Urgency
The treatment could cost between £1.5 million and £3 million, according to a Sanfilippo charity. Tammy had been applying for clinical trials that would have given Tate access to this therapy, but a delay in his official diagnosis meant the applications closed in August, just before the diagnosis was confirmed.
Tammy told Talk to the Press: "What hurts me is that I was applying for trials when they first put Sanfilippo on the table but they closed in August – just before we got the official diagnosis. If the diagnosis wasn't delayed then we could have got into one and life could be completely different which is incredibly frustrating but now I'm giving up applying for trials."
She added: "Once the FDA approves the treatment the only battle I have is money and the longer that takes the more Tate deteriorates. Tate is a spring chicken, he lives to climb, he runs like Usain Bolt, and I want to keep that."
Living in Hope and Denial
Tammy said she lives in a world of "hope and denial" because she must believe Tate will get the treatment. "I don't look past the end of the year because so much can change in just a few months but this treatment will allow me to look to our future again," she said.
The treatment is not yet approved in the US, but there is hope it will be by September of this year. Tammy is officially fundraising through Just4Children, a registered charity, and donations can be made to support the family.
