A nine-year old boy living with Duchenne muscular dystrophy has been offered a life-line in a letter from Scotland’s First Minister.
Michael Young was thrilled to receive the surprise letter from Nicola Sturgeon confirming that he will receive breakthrough Duchenne drug, Translarna.
It comes after his family launched a major campaign, which was backed by 150,000 people in Scotland, to get the drug approved for children with the life-shortening disease.
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 - Michelle Young
Michael and his mum and dad met with First Minister Nicola Sturgeon in June this year after the Scottish Medicine Consortium (SMC) decided in April not to approve the drug to treat the muscle wasting condition.
SMC’s claim that there was too much uncertainty over the benefits of Translarna to justify its costs was challenged by Michael’s family.
Now, with Sturgeon’s support, the drug can be made available on the NHS through an Individual Patient Treatment Request (IPTR) agreement.
Translarna is the first drug of its kind to treat an underlying cause of Duchenne muscular dystrophy. Thanks to the First Minister’s intervention around five other boys in Scotland who are eligible for the drug can also expect to receive access.
For nearly two years, families, together with Muscular Dystrophy UK, campaigned determinedly to access Translarna.
The Young family and charity representatives met Sturgeon at Holyrood twice to make the case for its approval. During one meeting, Michael presented Nicola Sturgeon with the petition of over 150,000 signatures calling for her urgent help in securing the drug.
Duchenne muscular dystrophy is a severe muscle-wasting, life-limiting condition. Most boys will be in wheelchairs by their twelfth birthday, and few live beyond their late 20s.
It is hoped Translarna could keep eligible boys walking for longer, while also maintaining the prospect of accessing potential treatments of the future.
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 of 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.
The drug was approved in England earlier this year and the first eligible boys will receive the drug on the NHS within the coming weeks.
