Michael Rankin, 12, who is desperately waiting for access to the drug, said: “Every second they don’t get this through, one person might not be able to walk again”.
News Jennifer Hyland Reporter 04:30, 20 Apr 2025
Children with a rare form of muscular dystrophy have been promised access to a groundbreaking drug after the Sunday Mail highlighted their plight. Around 30 boys could have their lives extended by Givinostat, which slows progress of the killer disease.
Duchenne muscular dystrophy (DMD) patients in Scotland have not been able to get the drug – which is available in England – despite manufacturers offering it to health boards for free on an early access programme. However NHS Greater Glasgow and Clyde has now confirmed eligible kids will be given Givinostat.
It comes ahead of Health Secretary Neil Gray meeting families and their lawyer Aamer Anwar on Tuesday. They will tell the SNP minister children are in a race against time to combat the tragic muscle wasting condition – which has a life expectancy of just 26 – and demand a date for the first doses.
DMD patient Michael Rankin, 12, who is desperately waiting for access to Givinostat, said: “Every second they don’t get this through, one person might not be able to walk again”.
His mum Nicola Rankin, 38, from Haddington, East Lothian, added: “It is great news our boys can access the early access programme, but how long will we actually need to wait?
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“Time is muscle, and our boys do not have time to wait. They have known for six months about the early access programme this should now be a top priority to start asap.
“This is a fight we shouldn’t have had to have. Scotland need to do better. The quality of our children’s lives are on the line.”
About 200 children in Scotland have DMD, an incurable condition affecting boys.
It causes progressive muscle weakness, leading to the loss of mobility, heart and lung problems and early death.
There are thought to be less than 30 children suitable for the Givinostat early access scheme in Scotland, but to date health boards have failed to provide any patients with the drug.
Lawyer Aamer Anwar, who has threatened legal action if it is not delivered, welcomed the access while demanding urgency.
He said: “This is great news, but on Tuesday, Neil Gray must confirm the date and if all the boys will get the drug.
"We did not launch this campaign four weeks ago so that only some of the boys get the drug, whilst others are left watching in misery as their muscles waste away.
“First Minister John Swinney, in his letter to me last week, said they had to ensure access to Givinostat was ‘safe, fair and equitable’ – surely that must mean all boys across Scotland and within the next fortnight.”
We previously told how Mhairi and Michael Harvey, whose son Michael, seven, has DMD, were told by the boss of NHS Greater Glasgow and Clyde that despite Givinostat being free, administering it would create an “increase in workload”.
In contrast, eligible patients at the Leicester Royal Infirmary got a first dose in March.
The situation in Scotland appears to be changing after we highlighted how families were threatening legal action.
When contacted by the Sunday Mail, Gray said: “We want young people living with DMD to benefit from new medicines such as Givinostat and live longer, fuller lives.
“I will be meeting families next week to provide reassurance that Health Boards are working at pace towards making Givinostat available in a safe and equitable way.”
NHS Greater Glasgow and Clyde said: “We are aware that the manufacturer has offered the drug for use free of charge, and we are currently going through a process to make Givinostat available to eligible patients.
"We are also working with other regional centres throughout Scotland and health boards across the west of Scotland to develop a plan for provision of this drug.”
Gray faced a backlash after failing to respond to a plea to help DMD patients while finding time to post on social media about traffic lights, youth football and even a snap of himself in sunglasses during hot weather.
Duchenne UK’s CEO, Emily Reuben OBE, said: “If a medicine for a devastating disease like DMD works, everyone who could benefit deserves access.
“This has been a long time coming. We call on NHS Trusts and Boards which host specialist DMD clinics to follow Leicester Royal Infirmary’s example, and take part in the Givinostat early access programme. Time is muscle, and we can’t afford a delay.”
Every day takes DMD patients closer to losing the ability to walk, move, or even breathe.
Some kids who would have been eligible for Givinostat in Scotland when it was first made available last year will no longer be able to access it due to having lost the ability to walk.
The National Institute for Health and Care Excellence (NICE) in England and Scottish Medicines Consortium (SMC) are assessing whether to provide it more generally on the NHS at which point a charge would likely be introduced.
A pharmaceutical firm may offer a medicine free for a time before SMC assessment.
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The Scottish Government has said while implementation of new medicines is for individual health boards, Gray has asked the Chief Pharmaceutical Officer to look at how to overcome barriers stopping clinicians from providing access.
The CPO met pharmaceutical firm Italfarmaco S.p.A last week to confirm when they will make a submission to the SMC.
