First Minister to meet young Duchenne patient over vital drug

A nine-year-old boy with a rare muscle-wasting condition will meet the First Minister as his petition calling for access to vital medication has neared 100,000 signatures.

Nine year old Michael Young from Larbert near Falkirk outside the Scottish Parliament as he prepares to deliver a letter to First Minister Nicola Sturgeon at the Scottish Parliament Edinburgh, calling for NHS access to medication.

Michael Young, from Larbert, has been campaigning for Nicola Sturgeon to intervene after the Scottish Medicines Consortium (SMC) rejected the drug Translarna to treat Duchenne muscular dystrophy.

Most people with the condition are confined to a wheelchair before they turn 12 and few live beyond the age of 30.

Translarna is the first drug to treat the underlying cause of the condition, allowing patients to be able to walk for longer.

The Young family will meet Ms Sturgeon next week to make a last ditch plea for the drug, which is currently available on the NHS in England.

Michael has previously presented Ms Sturgeon with a letter calling for access to the drug for the five boys in Scotland eligible for treatment.

Michael’s mother Michelle Young, said: “For those just the other side of the border, the drug is still moving forward, while our hopes have been crushed.

“Nicola Sturgeon has agreed to meet us. Now we need her to act.

“We need her to help us find a way to make sure all the boys in Scotland, who could benefit from this drug, are able to get it. Time is running out for these children.”

Cormac Fegan, of Longniddry, East Lothian, became the first child in the UK to get access to the Duchenne muscular dystrophy (DMD) drug Translarna on the NHS, after a successful individual funding request to NHS Lothian.

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, said: “The overwhelming support from the public for Michael’s petition shows the widespread recognition that the SMC got it wrong.

“The public clearly see that for boys like Michael, accessing Translarna could make such a difference to their lives. We are talking about just five boys who desperately need this new treatment.

“The fact that boys just like Michael can access the drug in England means families will feel forced to uproot their lives, leaving their support networks and moving across the border.

“We urge the First Minister to work with us, the SMC, and the makers of the drug to find a solution.”

The SMC has met with the drug company, which indicated it may make another submission.