Yesterday 05:38 PM
(This post was last modified: Yesterday 10:41 PM by C C.)
Not a knock on NHS, though, since most health insurance plans in the US don't cover costs in other countries, either. You have to buy travel or international health insurance before the trip or sojourn.
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Brit boy, 5, with rare dementia could be treated but at massive cost in millions
https://www.mirror.co.uk/news/uk-news/de...d-37855607
Tate McDaid was four when he was diagnosed with Sanfilippo Syndrome - a rare genetic disorder which progressively damages the brain, causing children to lose abilities they have already learned including speaking, walking and eventually eating independently. The disorder affects just over 100 children in the UK, and is often referred to as 'childhood dementia' or 'childhood Alzheimer's'.
The first gene therapy to treat the condition was approved in the US in September, but at a cost of £2.95 million per child. Tate's mum, Tammy, has been left desperately trying to raise enough funds to get her son the ground-breaking treatment. Tammy, from Swansea, said it was a "battle of time" as Tate is turning six this month and the disorder progresses more rapidly after the age of five.
The mum said she was excited when the treatment initially got approved, but that "reality hit hard the next day" after seeing that the US price had been set at $3.95m. "We understand what they are charging for American citizens, but they've got health insurance," she told the BBC. "What is it for us in the UK? Not even just the UK, but everyone else in the world looking for this treatment."
Tammy has been in touch with Ultragenyx, who developed the treatment, to find out how families without health insurance in the US will cover costs.
There is currently no cure for Sanfilippo Syndrome Type A. Tammy says she is "petrified" that the family don't have enough time as Tate has "already shown signs of cognitive decline". She explained: "The older he gets the less the treatment will work and the more ability he will lose in the process. I feel like we are at a turning point and I'm petrified.
"If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away," Tammy added. She began fundraising before the gene therapy was approved by the US Food and Drug Administration. So far, Tammy has raised just over £200,000 - but this only covers around 6% of the estimated cost.
"Unfortunately, we're in a world where we need millions, not hundreds of thousands," she said. Tammy said Tate is "the happiest little man" who is "blissfully unaware" of his health issues. "He doesn't speak, he's never spoken. But his mobility is everything to him and that is why I'm fighting for it," she said.
Ultragenyx said it was seeking a global regulatory strategy and FDA approval to bring the treatment to families around the world. The company added: "We also understand the urgency families affected by Sanfilippo syndrome Type A face in identifying treatment options and why some may consider travelling to the US to access gene therapy.
"We recommend families work with their child's healthcare provider to understand what potential access pathways may exist before assuming this is their only option." Noah's Ark Children's Hospital in Cardiff is currently liaising with officials in the US on the family's behalf.
- - - - - - - - -
Brit boy, 5, with rare dementia could be treated but at massive cost in millions
https://www.mirror.co.uk/news/uk-news/de...d-37855607
Tate McDaid was four when he was diagnosed with Sanfilippo Syndrome - a rare genetic disorder which progressively damages the brain, causing children to lose abilities they have already learned including speaking, walking and eventually eating independently. The disorder affects just over 100 children in the UK, and is often referred to as 'childhood dementia' or 'childhood Alzheimer's'.
The first gene therapy to treat the condition was approved in the US in September, but at a cost of £2.95 million per child. Tate's mum, Tammy, has been left desperately trying to raise enough funds to get her son the ground-breaking treatment. Tammy, from Swansea, said it was a "battle of time" as Tate is turning six this month and the disorder progresses more rapidly after the age of five.
The mum said she was excited when the treatment initially got approved, but that "reality hit hard the next day" after seeing that the US price had been set at $3.95m. "We understand what they are charging for American citizens, but they've got health insurance," she told the BBC. "What is it for us in the UK? Not even just the UK, but everyone else in the world looking for this treatment."
Tammy has been in touch with Ultragenyx, who developed the treatment, to find out how families without health insurance in the US will cover costs.
There is currently no cure for Sanfilippo Syndrome Type A. Tammy says she is "petrified" that the family don't have enough time as Tate has "already shown signs of cognitive decline". She explained: "The older he gets the less the treatment will work and the more ability he will lose in the process. I feel like we are at a turning point and I'm petrified.
"If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away," Tammy added. She began fundraising before the gene therapy was approved by the US Food and Drug Administration. So far, Tammy has raised just over £200,000 - but this only covers around 6% of the estimated cost.
"Unfortunately, we're in a world where we need millions, not hundreds of thousands," she said. Tammy said Tate is "the happiest little man" who is "blissfully unaware" of his health issues. "He doesn't speak, he's never spoken. But his mobility is everything to him and that is why I'm fighting for it," she said.
Ultragenyx said it was seeking a global regulatory strategy and FDA approval to bring the treatment to families around the world. The company added: "We also understand the urgency families affected by Sanfilippo syndrome Type A face in identifying treatment options and why some may consider travelling to the US to access gene therapy.
"We recommend families work with their child's healthcare provider to understand what potential access pathways may exist before assuming this is their only option." Noah's Ark Children's Hospital in Cardiff is currently liaising with officials in the US on the family's behalf.
