There's finally a treatment for my 5-year-old son's dementia, but it costs US$4 million

Image source, Tammy McDaidPhoto caption, Tate's mother, Tammy McDaid, needs US$4 million for a treatment that will save her son's life. Author, Danielle HerbertAuthor's title, BBC WalesDate of publication 33 minutes agoReading time: 5 minThe mother of a five-year-old boy with a rare form of dementia has to pay nearly US$4 million for the treatment that will save his life. Tate McDaid, from Swansea, Wales, was diagnosed with Sanfilippo syndrome, a form of dementia affecting just over 100 children in the UK, when he was four years old. In September, the first gene therapy to treat this disease in children was approved in the United States. His mother, Tammy, said she spent "months praying" for the treatment to be approved, but discovered it will cost nearly US$4 million per child, and it is unclear how families outside the United States will be able to finance it. Welsh doctors contacted their American counterparts to see what possible access routes existed for Tate."I was thrilled when the treatment was initially approved, but the next day reality hit me hard," said Tammy. That was when she read that the price had been set at US$3.95 million. Tammy contacted Ultragenyx, the company that developed the therapy, hoping to understand how families living outside the North American country who do not have health insurance there could cover the costs. Skip Most read and continue readingMost readEnd of Most read"We understand what they charge US citizens, but they have health insurance," she added."What does this mean for us in the UK?" she said. "Not just in the UK, but for everyone seeking this treatment." When explaining how the drug FAYUVI, approved in the United States, would work for families outside that country, Ultragenyx stated: "We cannot offer a single estimate of the total cost of care for an international patient, as these costs depend on each patient's specific medical needs and the institution providing their care."What is childhood dementia?Sanfilippo syndrome type A, also known as mucopolysaccharidosis type III (MPS III), causes the gradual loss of abilities such as walking and eating. Due to its neurodegenerative nature, it is often called "childhood Alzheimer's" or "childhood dementia." According to the Society for Mucopolysaccharide Diseases (MPS Society), a charity that helps children living with Sanfilippo syndrome, around 140 children in the UK have this condition. There is currently no cure for the syndrome, but the new drug could delay its onset and allow Tate to stay active for longer. The condition can progress more rapidly after the age of five, which is why Tammy says that raising enough money for her son to receive the treatment, which is not yet available in his country, "is a real race against time.""If a millionaire showed up tomorrow and gave us the money, we would go to the United States immediately." In Tate's case, who turns six this November, Tammy does not believe time is on their side."Tate is already showing signs of cognitive decline," she added."The older he gets, the less effective the treatment will be and the more abilities he will lose in the process." "I feel like we are at a turning point and I am terrified." Image source, Tammy McDaidPhoto caption, Tammy said the new drug could help her son maintain his mobility for longer. Tammy describes Tate as "the happiest little man in the world" who "completely ignores" his problems, and explained how important mobility is to him."He doesn't speak, he has never spoken. But his mobility is everything to him and that is why I am fighting for it," she said. The mother began fundraising before the US Food and Drug Administration approved the new drug, and now has more than US$215,000. But this only covers about 5% of the estimated total cost of the treatment."Unfortunately, we live in a world where we need millions, not hundreds of thousands," she added."I just hope that, by some miracle, the pharmaceutical company contacts us again and tells us they can make it a bit cheaper, but even then it will cost millions." Image source, Tammy McDaidPhoto caption, Tate's mother, Tammy, has raised more than US$215,000 so far. Ultragenyx stated: "We are pursuing a global regulatory strategy, and the FDA approval is an important first step in our long-term goal of offering this treatment option to families around the world.""We also understand the urgency with which families affected by Sanfilippo syndrome type A face the search for treatment options and why some may consider traveling to the United States to access the gene therapy.""We recommend that families reach out to their child's healthcare provider to understand what possible access routes exist before assuming this is their only option." Download the new BBC World Service app. Choose BBC News Mundo and receive news alerts, documentaries and analysis, all in one place. Important: the new app is not available in the UK or the United States. You can also follow us on YouTube, Instagram, TikTok, Facebook, X and on our WhatsApp channel. And don't forget to subscribe here to our newsletter to receive a special selection of our best content every Friday. This article was originally written in English and we used an artificial intelligence tool to translate it. A BBC journalist reviewed the text before publication. More information on how we use AI. Discover moreSkip Video and continueVideoEnd of VideoMore top stories