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The mother of a five-year-old Welsh boy with a rare type of dementia will have to pay around R20.3 million for treatment that could save his life.
Tate McDaid, from the city of Swansea, Wales, was diagnosed with Sanfilippo Syndrome — a type of dementia he shares with just over a hundred British children — when he was 4 years old.
In September of this year, the first gene therapy — that is, one that modifies genetic material — was approved in the United States to treat the disease.
Tate's mother, Tammy, says she spent "months praying" for the treatment to be ratified, until she discovered the high cost of doing so, as well as doubts about the possibilities of access to it for people who don't live in the US.
Welsh doctors have already reached out to American colleagues for information on how Tate could receive the medicine.
“I was thrilled when they initially approved the treatment, but the next day reality hit me hard,” said Tammy.
That's when she read that the price of the medicine was exactly US$3.95 million, equivalent to around R$20.3 million.
Tammy contacted Ultragenyx, the US company that developed the therapy, in the US, hoping to understand how families in other countries, without medical insurance, could cover the costs.
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“We understand that they charge this amount to US citizens: they have medical insurance,” says Tammy. “But what does this mean for us in the UK and for everyone seeking this type of treatment?”, he adds.
In explaining how the FAYUVI drug would work for families outside the U.S., Ultragenyx stated, "We cannot provide a single estimate of the total cost of treatment for an international patient, as it depends on each patient's specific medical needs and insurance plan."
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Sanfilippo Syndrome type A, also known as Mucopolysaccharidosis type 3 (MPS III), causes a gradual loss of physical abilities, such as walking or running. Because it is neurodegenerative, it is often called "childhood Alzheimer's" or "childhood dementia".
According to the National MPS Society, an NGO created in the 1970s in the USA focused on mucopolysaccharidoses, such as Sanfilippo Syndrome, around 140 British children currently live with it.
In Brazil, data on the syndrome is scarce, but the MPS Brasil Network estimates a minimum incidence of 1 case for every 100,842 births in the country.
There is still no cure, but the new treatment could delay its onset, allowing children like Tate to remain active for longer.
The condition can progress more quickly after age 5, so Tammy says raising enough money for her child to receive treatment "is a real battle against time."
“If a millionaire came along tomorrow and gave us that money, we would go to the US immediately,” she says.
But in the case of Tate, who turns 6 this month, Tammy doesn't believe time is on her side. “He is already showing the first signs of cognitive deterioration,” he says. "The older he is, the less effective the treatment will be and the more capabilities he will lose."
"I feel like we're at a tipping point. I'm terrified," she adds.
Tammy describes Tate as the "happiest little man in the world" who "completely ignores his condition." It also notes that although he never talks about it, mobility is very important to him. "That's why I'm fighting for her."
The mother began raising funds before the Food and Drug Administration (FDA), the body responsible for validating medicines in the US, approved the new treatment. She already has about $215,000, but that only covers 5% of the cost of her treatment.
"I hope that, by some miracle, the company comes back to us and says they can give us a discount — which will still cost millions."
Ultragenyx stated, "We are pursuing a global regulatory strategy. 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 seek treatment options and why some consider traveling to the US to access this therapy."
"We recommend that these families look into their medical insurance to understand what avenues of access exist before assuming that this [asking the company for help] is the only option."
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