A Dunfermline dad says he will move his family to the USA after his six-year-old son was denied a free drug that could prolong his life.
A rare disease affecting mostly young boys, Duchenne Muscular Dystrophy (DMD) is a progressive muscle disorder which leaves most of those diagnosed with the condition in a wheelchair by the age of 12.
Jamie Tierney was diagnosed with the disease aged three.
With no cure, the Tierney family, including mum Bobbie and dad Jamie senior, were simply told to go home and enjoy what time they had after the shock diagnosis.
But Jamie’s dad told The Courier this “didn’t sit right” and they decided to act.
‘We were told to go home and enjoy our son’
Mr Tierney said: “It was a shock to our family.
“We were more or less told to go home and enjoy our son.”
It was at this point Mr Tierney started doing his own research looking at new treatments.
While there is no cure the family discovered a number of promising new drugs.
Family travel to USA, Colombia and Dubai to find treatment
It is a journey that has taken them to Ireland, Colombia, the USA, Mexico and Dubai – each trip costing around £20,000.
One drug they learned about is givinostat, a treatment for DMD which can slow down the progression of the disease that was licensed in the UK last March.
But progress in rolling the drug out has stalled while medics assess its cost-effectiveness.
But Mr Tierney says his son does not have time to wait with his condition worsening every week.
While the drug is available to children in England, NHS Lothian – who provide neurology care for Fife patients – have told Jamie’s mum and dad they do not currently have the infrastructure to make it available.
“It was made available on an early access programme from December 2024 but no boy in Scotland has received it.
‘Days are crucial’
“It is an oral drug which would be given daily. We’ve been told that the NHS doesn’t have the infrastructure. We were furious. It’s also being paid for by the company, so there’s no cost to the NHS,” Jamie’s dad said.
He added: “There’s children who have come off their feet in the time this has been available.
“Days, weeks, everything is crucial.”
After spending £80,000 a year on new treatments abroad, Mr Tierney says he will soon put the family home on the market and explore a move to the USA.
“Over the last three years, watching the development here compared to other countries, we’ve found it incredibly frustrating.
“If it’s so difficult to get an oral drug out, what sort of chance have we got when better drugs (are made available).
“We felt let down. Because it is a minority of boys they just don’t seem bothered.”
Dunfermline and Dollar MP Graeme Downie is supporting the family.
Children denied DMD drug as ‘givinostat lies on a shelf’
He has called for the Scottish Government to accelerate the prescription of the drug.
Other families of children with DMD are also backed by campaigning solicitor Aamer Anwar.
Mr Anwar said: “It is cruelty beyond belief that these young boys are being denied a medication that would increase their quality of life.
“Health secretary Neil Gray needs to act now and do the right thing before it’s too late.”
Mr Downie told The Courier: “The NHS in England has overcome barriers and started treatment but that seems beyond the capability of the Scottish Government.”
“Every day that givinostat lies on a shelf, hundreds of children across Scotland lose a little bit more of their strength and health.
“That is the consequence of focusing on bureaucracy rather than people, and it will be the SNP’s legacy.”
NHS Lothian’s medical director, Dr Tracey Gillie, said medicines under evaluation are “not routinely prescribed”.
She added: “There are processes in place to provide earlier access to medicines in the interim period and patients and their circumstances are considered on an individual basis.”
A Scottish Government spokesman said: “It is for individual health boards to consider the introduction of any new medicine within an existing or new care pathway, even when offered free of charge.”
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