image copyrightTammy McDaid
The mother of a five-year-old boy with a rare form of dementia has to pay almost $4 million for life-saving treatment.
Tate McDaid, from Swansea, Wales, was diagnosed with Sanfilippo syndrome, a form of dementia that affects 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 found out it will cost about $4 million per child, and it's unclear how families outside of U.S. soil will be able to finance it.
Welsh doctors contacted their American counterparts to see what possible avenues existed for Tate.
"I was excited when they initially approved the treatment, but the next day reality hit me hard," Tammy said.
That's when he read that the price had been set at $3.95 million.
Tammy contacted Ultragenyx, the company that developed the therapy, hoping to understand how families living outside the country who don't have health insurance there could cover the costs.
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"We understand what they charge American citizens, but they have health insurance," he added.
"What does this mean for us in the United Kingdom?" he said.
"Not just in the UK, but for everyone seeking this treatment."
Explaining how the US-approved drug FAYUVI would work for families outside the United States, Ultragenyx stated: "We cannot offer a single estimate of the total cost of care for an international patient, as these costs depend on the specific medical needs of each patient and the institution providing care."
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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 Mucopolysaccharide Diseases Society (MPS Society), a charity that helps children living with Sanfilippo syndrome, around 140 children in the UK suffer from the 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 age five, so Tammy says raising enough money for her son to receive treatment that is not yet available in her country "is a real battle against time."
"If a millionaire showed up tomorrow and gave us the money, we would go to the United States immediately."
In the case of Tate, who turns six this November, Tammy doesn't think time is on her side.
"Tate is already showing signs of cognitive decline," he added.
"The older you get, the less effective the treatment will be and the more capabilities you will lose in the process."
"I feel like we're at a tipping point and I'm terrified."
Tammy described Tate as "the happiest little man in the world" who "completely ignores" her 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's why I'm 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 $215,000.
But this only covers about 5% of the total estimated cost of treatment.
"Unfortunately, we live in a world where we need millions, not hundreds of thousands," he added.
"I'm just hoping that by some miracle the drug company will get back to us and tell us they can make it a little cheaper, but it will still cost millions."
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 gene therapy."
"We encourage families to reach out to their child's healthcare provider to understand what potential avenues of entry exist before assuming this is their only option."
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. Learn more about how we use AI.
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