Parents devastated as breakthrough drug denied funding

Michael Young with his letter outside the Scottish Parliament
Michael Young with his letter outside the Scottish Parliament

Heartbroken parents of a boy with a life-limiting muscle-wasting illness say they will appeal a decision to deny him access to a breakthrough drug.

Michelle and Justin Young from Larbert said they were devastated when the Scottish Medical Consortium (SMC) confirmed this week it would not approve funding for Translarna, the first therapy available to treat an underlying cause of Duchenne muscular dystrophy.

Their son Michael (9) hit the headlines earlier this year when he wrote to Nicola Strugeon explaining how the drug could help him.

Michael is thought to be one of five boys in Scotland eligible for the drug which slows down progression of the condition while the children are still able to walk.

Michelle said: “We are devastated by the SMC’s decision. It’s already available in many European countries. Yet here, boys like Michael will continue to lose the ability to walk before the age of 10, to rely on steroids with severe side effects, go through painful surgery, loss of muscle function without any reprieve. How can we possibly accept that, despite medicine being available, nothing will be done?”

The SMC has said it did not believe the treatment’s cost was justified in relation to its health benefits.