The mum of a five-year-old boy with a rare form of dementia has to pay almost £3m for his life saving treatment.
Tate McDaid, from Swansea, was diagnosed with Sanfilippo Syndrome – a form of dementia that just over 100 UK children have – when he was four.
In September, the first gene therapy to treat the condition in children was approved in the US.
His mum Tammy said she spent "months praying" for its approval but discovered the treatment is to set cost £2.95 million per child, with it unclear how families outside the US can fund this.
Health officials from Wales are currently talking to those in the US to see what potential access pathways there are for Tate.
"I was excited when the treatment initially got approved but then reality hit hard the next day," Tammy said.
This was when she read the US price for the one-time treatment, external had been set at $3.95 million.
Tammy contacted Ultragenyx, who developed the treatment, in the hope of understanding how families outside the US, who do not have health insurance there, will cover the costs.
"We understand what they are charging for American citizens, but they've got health insurance," she added.
"Not even just the UK, but everyone else in the world looking for this treatment."
Explaining how the US approved drug, FAYUVI, would work for families outside the US, Ultragenyx said: "There is not a single estimate we can provide for the total cost of care for an international patient, as those costs would depend on the patient's individual medical needs and the treating institution."
What is childhood dementia?The Society for Mucopolysaccharide Diseases, external, a charity which helps children living with Sanfilippo Syndrome, said about 140 children in the UK have the condition.
There is currently no cure for Sanfilippo Syndrome Type A, also called Mucopolysaccharidosis type III (MPS III), but the new drug could delay its onset, and allow Tate to remain active for longer.
The condition, described as "childhood Alzheimer's" or "childhood dementia" due to its neurodegenerative nature, causes the gradual loss of abilities, such as walking and eating.
It can progress more rapidly after the age of five, which is why Tammy said "it's really a battle of time" for her to raise enough money to get her son the treatment that is not available yet in the UK.
She said: "If a millionaire turned around tomorrow and gave us the money, we'd go to the US straight away."
With Tate turning six this month, Tammy doesn't believe time is on their side.
"Tate is already shown signs of cognitive decline," she added.
"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."
Describing him as "the happiest little man" who is "blissfully unaware" of his problems, Tammy explained how important Tate's mobility is to him.
"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.
Tammy began raising money before the new drug was approved by the US Food and Drug Administration and now has more than £160,000.
But this covers only about 5% of the total estimated cost of the treatment.
"Unfortunately, we're in a world where we need millions, not hundreds of thousands," she added.
"I'm just really hoping by some miracle the pharmaceutical company eventually come back to us and say they can do it for a little bit cheaper, but it's still going to be in the millions."
Ultragenyx said: "We are pursuing a global regulatory strategy, and FDA approval is an important first step in our long-term goal of bringing this treatment option to families around the world.
"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."
Staff from the Noah's Ark Children's Hospital in Cardiff are currently discussing this with officials in the US on the family's behalf.
Fox Style News Syndicate. Sourced from broadcast reporting: Read Full Source Coverage.