The mother of an eight-year-old boy with Sanfilippo syndrome type A is racing to raise £2.95 million for a newly approved gene therapy in the US. Georgia Nonas, 29, from Gateshead, launched a fundraising appeal after the US Food and Drug Administration (FDA) approved UX111, the first gene therapy for the condition.
Diagnosis and early signs
Cody Carroll was a healthy toddler until he began experiencing recurring ear infections and hearing loss at age two, followed by an autism diagnosis. A medical professional later noted his "coarse features," including thick eyebrows and a prominent forehead, which prompted further testing and confirmed Sanfilippo syndrome in July 2021.
The rare disease, known as childhood dementia, causes severe intellectual disability and typically leads to death in the teenage years. Cody's body lacks an enzyme needed to process waste, which builds up on his spinal cord and brain, progressively damaging his ability to communicate, eat, and walk.
Treatment hope and fundraising
Georgia said the FDA approval has given the family "more hope than we've ever had," but the treatment costs £2.95 million and is not yet available in the UK. She noted that discussions about UX111 in the UK are not due until April, and it could be years before it is considered.
A GoFundMe page has raised £16,000 so far. Georgia plans to fundraise through a charity and apply for medical grants, saying, "We just can't sit back and wait and wonder what might have happened if we'd acted sooner."
Impact on family and future
Cody's condition has progressed, leaving him reliant on a wheelchair, unable to swallow solid foods, and requiring daily painkillers and a sleeping aid. He also has epilepsy. Georgia described the emotional toll: "I'm watching my child fade away every single day, and it's enough to break a person."
She remains hopeful about the treatment's potential to slow the disease's progression and is advocating for earlier diagnosis and access. "For our family, this approval doesn't mean the fight is over; it means, for the first time, that we have something real to fight for," she said.
