FAMILIES are hoping a ground breaking drug which helps children with a rare condition to walk will be approved for use on the NHS in Scotland today.
The treatment, Translarna, is the first treatment ever to address the underlying cause of Duchenne muscular dystrophy and it helps young sufferers - who often depend on wheelchairs by the age of eight - to stay on their feet for longer.
While the drug has been available to patients in other European countries such as France, Spain and Germany for more than a year, families in the UK have been waiting 18 months to hear if the NHS will prescribe the treatment.
Now the Scottish Medicines Consortium (SMC), which evaluates new drugs to decide if they should be available on the NHS, is due to announce their decision.
Michelle Young, whose son Michael suffers from Duchenne muscular dystrophy, said: "It’s been a long wait for us as a family to find out the SMC’s answer on Translarna."
Earlier this year nine-year-old Michael, from Larbert, wrote a heart-felt letter to First Minister Nicola Sturgeon appealing for her help to ensure patients like himself could have the treatment. He had been given the drug as part of a trial but was worried what would happen as the trial had finished. He told Ms Sturgeon: "If my medicine is stopped I will need a wheelchair."
Mrs Young said: "We are so proud of Michael for writing to the First Minister and making the opportunity to tell her face-to-face about what the drug would mean for him. I really do hope she understood what impact the drug could have on all of our lives.
“Michael doesn’t let his frustrations show, but being unable to keep up with friends or join the football team, at just nine years old, is very tough."
She described Translarna as the first piece of a jigsaw puzzle in treating his condition: "It is putting off some very difficult challenges for Michael, but, also, it is buying time. The clinical trials taking place and the drugs that are being developed are for boys who are still walking. Once the ability is lost, so is the chance to benefit from treatments that could transform Michael’s future.
“The cost to the NHS of ignoring the reality of life with Duchenne is great."
Duchenne muscular dystrophy is an inherited genetic condition which mostly affects boys. The genetic mutations cause changes in the muscle fibres which interfere with the way muscles work, leading to increasing disability. When suffers reach their teens and twenties the problem can begin to affect their heart and chest with few patients surviving beyond their thirties.
However, new treatments being developed for the condition are giving families hope.
Cormac Fegan, aged four from Longniddry in East Lothian, is the only child in the UK to receive Translarna on the NHS after a special request for funding was made to his health board.
Gary Fegan, his father, said: “It’s my greatest hope that the SMC will approve Translarna for Cormac and other children like him. Whilst Cormac has only been on Translarna for five months, the difference we’ve seen in him, not only physically, but also in terms of his behaviour, has been incredible. There’s nothing to suggest that this won’t continue.
“We are treating children with Duchenne with steroids, which often have terrible, irreversible side-effects, in the hope that, when a drug like Translarna becomes available, they still have enough functioning muscle to benefit.
“If Translarna is rejected by the SMC then what hope does this give families that future drugs will be approved and why should families hold out any hope at all?”
Muscular Dystrophy UK say families face a race against time for access to the drug, as children with Duchenne muscular dystrophy must still be able to walk to be eligible for the treatment. The charity says if SMC rejects the drug, some boys could lose the chance to take it entirely. A decision is due at 2pm on Monday April 11.
Why are you making commenting on The Herald only available to subscribers?
It should have been a safe space for informed debate, somewhere for readers to discuss issues around the biggest stories of the day, but all too often the below the line comments on most websites have become bogged down by off-topic discussions and abuse.
heraldscotland.com is tackling this problem by allowing only subscribers to comment.
We are doing this to improve the experience for our loyal readers and we believe it will reduce the ability of trolls and troublemakers, who occasionally find their way onto our site, to abuse our journalists and readers. We also hope it will help the comments section fulfil its promise as a part of Scotland's conversation with itself.
We are lucky at The Herald. We are read by an informed, educated readership who can add their knowledge and insights to our stories.
That is invaluable.
We are making the subscriber-only change to support our valued readers, who tell us they don't want the site cluttered up with irrelevant comments, untruths and abuse.
In the past, the journalist’s job was to collect and distribute information to the audience. Technology means that readers can shape a discussion. We look forward to hearing from you on heraldscotland.com
Comments & Moderation
Readers’ comments: You are personally liable for the content of any comments you upload to this website, so please act responsibly. We do not pre-moderate or monitor readers’ comments appearing on our websites, but we do post-moderate in response to complaints we receive or otherwise when a potential problem comes to our attention. You can make a complaint by using the ‘report this post’ link . We may then apply our discretion under the user terms to amend or delete comments.
Post moderation is undertaken full-time 9am-6pm on weekdays, and on a part-time basis outwith those hours.
Read the rules hereLast Updated:
Report this comment Cancel