TIME is running out for a young Donegal schoolboy whose entire mobility is at risk because of a rare genetic condition, his family has warned.
Aaron Langan, a nine-year-old from Laghey, has terminal illness Duchenne muscular dystrophy (DMD), an incurable condition that causes his muscles, including his heart, to slowly stop working, and can lead to scoliosis.
His lower body is now so weak that he needs a wheelchair full-time but a new drug would stop DMD overtaking his upper body as well.
However, the cost of the drug Givinostat – which was approved by the European Medicines Agency (EMA) last June – has not been sanctioned by Ireland’s Health Services Executive (HSE) nearly a year later amid a cost-benefit analysis review.
His family warned that “time is muscle” and fear Aaron could lose more strength and miss out on the drug – as it is only licensed for those who are able to move.
Aaron’s mum Sinead said: “You can just imagine these people, sitting around a table, asking, ‘Would it be value for money? Should we let his mobility deteriorate more? Or should we [provide] the drug for a couple of years so that he can have a wee bit more mobility and be happier with more quality of life?’
“That is so frustrating. We are just so disappointed. There are kids on this island [in the North] who are receiving this drug.
“It is just so frustrating that we are still waiting on it since last June. The HSE is waiting for the company to come back with a price so negotiations can begin.”
She added: “Things feel like they are going down rapidly. We just hope that we can get this drug.”
There are 150 children in Ireland with Duchenne muscular dystrophy (DMD).
When the EMA ruled that Givinostat is a treatment for children who are at least six years old, some parents protested outside the Dáil to demand that the HSE cover the cost of the miracle drug.
Figures have put the cost between €92,000 and €348,000 per child, depending on the patient’s weight.
A health report the estimated that costs of the drug for the HSE could run to between €26m and €58m over five years.
She said: “We know the drug is not a cure. We just want to give Aaron the best quality of life. The guilt is unbelievable. We are just heartbroken.”
Donegal County Cllr Martin Harley spoke out in recent days to highlight Aaron’s case, while senator Manus Boyle is to be raise awareness of his plight this week.
Cllr Harley said: “This is about getting pain relief and some quality of life for Aaron.