The family of a young boy with a rare medical condition are celebrating a campaign victory after a letter from the First Minister confirmed he will benefit from a breakthrough drug earlier ruled out in Scotland.
Michael Young, nine, and his family met Nicola Sturgeon in June after the Scottish Medicines Consortium (SMC) refused to approve Translarna to treat the muscle-wasting condition Duchenne muscular dystrophy.
The SMC said in April there was too much uncertainty about the overall benefits in relation to its cost.
The family organised an online petition calling for a rethink of the decision which received the backing of more than 150,000 people.
A personal letter from Ms Sturgeon confirmed Michael and five other boys eligible for the drug will receive it through the individual patient treatment request agreement.
Most of the 100 boys born each year with with Duchenne will be in wheelchairs by their 12th birthday and few live beyond their late 20s, the charity Muscular Dystrophy UK said.
It is hoped Translarna could keep eligible boys walking for longer while also maintaining the prospect of accessing potential treatments of the future.
There are around currently 2,500 people living with the condition in the UK.
Michelle Young, Michael’s mother said: "To have our lovely quiet boy rewarded for all his brave and tireless campaigning was just amazing, for him and our family.
"Knowing that we no longer have to worry about Michael receiving his medication is a tremendous relief.
"Any extra time Michael spends on his feet is just invaluable.
“We are profoundly thankful for the time Nicola Sturgeon spent with us.
"To receive this letter from her was just a wonderful surprise and a much needed lifeline.
"It’s so special knowing Michael will be given vital medicine and can just get on with enjoying his childhood again.”
Robert Meadowcroft, chief executive, Muscular Dystrophy UK said: “Michael and his family have displayed such incredible determination, commitment and patience with his dedicated campaign.
"To receive this surprise letter from the First Minister is an absolutely well-deserved end to our battle to access Translarna.
“We would like to say a big thank you to Nicola Sturgeon, who has been focused and engaged throughout this campaign.
"It is entirely admirable that she twice met with the Youngs, and our charity to hear our concerns. To carry out such a vital intervention at a time of concern and uncertainty for the boys’ families is exceptional and greatly appreciated. It means everything to all those in Scotland who have battled so hard.
“After such impressive, spirited campaigning, the Youngs and affected families across Scotland will rightly enjoy the relief this letter brings.
"Meanwhile, in England, the first eligible boys for Translarna will receive the drug on the NHS within the coming weeks.”
In 2015 Cormac Fagan from East Lothian became the first child in the UK to receive NHS funding for Translarna, which has been available in several European countries since it was approved by the EU in August 2014.
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