Campaigners’ hopes were dashed when the Scottish Medicines Consortium (SMC) rejected Translarna yesterday, which is the first available drug to address the underlying cause of Duchenne muscular dystrophy (DMD).
The inherited condition, which mainly affects boys, causes muscles to waste over time so sufferers are often reliant on a wheelchair from a young age.
The heart and breathing muscles are also affected, meaning few people with the condition survive beyond their 30th birthday.
Michael Young, nine, who has the condition, wrote to the First Minister in January about the drug, which he has been given as part of a clinical trial. His family is now concerned it may be withdrawn when the trial ends.
Mother Michelle, of Larbert, near Falkirk, said: “We are devastated by the SMC’s decision not to approve Translarna in Scotland. It is already available in many European countries. Yet here, boys like Michael will continue to lose the ability to walk before the age of ten, to rely on steroids with their severe side effects, go through painful surgery, loss of muscle function, ventilation, without any reprieve.
“How can we accept that, despite medicine being available, nothing will be done?”
The charity Muscular Dystrophy UK has called on Nicola Sturgeon to intervene.
Chief executive Robert Meadowcroft said: “Translarna could give eligible boys precious time to be able to walk for longer, helping to delay the progression of the condition.
“For a young person with a shorter life expectancy, facing the acute psychological, physical and practical challenges of a progressive condition, this is of immeasurable value.”
Professor Jonathan Fox, chairman of SMC, said: “Despite powerful and moving testimonies given by patient groups and clinicians for this medicine, there was uncertainty around the overall health benefits in relation to the medicine’s cost and the committee was not satisfied that it would represent a good use of NHS resources.”
The SMC accepted three medicines, including a secondary breast cancer drug called Afinitor which can delay chemotherapy by six months.