A north-east family have expressed their devastation over the news that their daughter is not eligible for life-changing medicine.
Allyson and Stuart Townhill, of Johnshaven, had hoped that new drug Nusinersen – known as Spinraza – would provide relief for the family.
Roy and Lucy Townhill, aged nine and five respectively, were both diagnosed with the rare degenerative neuromuscular condition called Spinal Muscular Atrophy (SMA) Type 3, following years of diagnostic tests.
However, after a review by the Scottish Medicine Consortium (SMC), it was revealed that only people living with SMA Type 1 will be eligible.
Mum Allyson said: “Our son is strong but sadly Lucy is very weak and it’s so upsetting watching your child slowly become less able and suffer more.
“When we heard the SMC was starting a review for Spinraza, our spirits were immediately lifted.
“We know it’s not a cure, but it could give Lucy the chance to do the simple things other children take for granted.”
Though they have been supported by their community in Johnshaven, the part-time school teacher Allyson is finding the decision difficult to come to terms with.
She added: “We are so concerned when Lucy gets sick.
“It could hospitalise her. She is getting weaker and weaker as time goes by.
“Everyone should be given the same opportunity. I know it’s crazy expensive but we would love Lucy to have a more independent life —just to enjoy being a child.”
SMC chairman Dr Alan MacDonald said: “Unfortunately, the evidence presented suggested that Nusinersen was substantially less cost effective when used in types 2 and 3 and therefore could not be accepted by the committee.
“These are incredibly difficult decisions.
“The medicine is extremely expensive and there is a need to consider all those who need treatment by NHS Scotland.”
Robert Meadowcroft, the chief executive of Muscular Dystrophy UK, said: “It is simply impossible to comprehend why Spinraza isn’t available for all those who could benefit from it as it’s the only treatment option for these children.”