A DEESIDE mum fears pioneering treatments may never reach her son in time.
Debbie Vaughan’s son Harry, six, has congenital muscular dystrophy which causes muscles to weaken and waste over time, leading to increasing disability and eventually, life-threatening health issues.
Findings of a six-month parliamentary inquiry into the provision of future high-cost medicines for rare diseases reveal potential treatments for progressive genetic conditions such as Duchenne muscular dystrophy are finally in clinical trials.
However, parents fear that a race to get their children treated in time may be lost due to funding issues and red tape.
Mrs Vaughan, of Connah’s Quay, gave evidence to the parliamentary inquiry and is anxious at the pace of the research into treatment for her son’s condition.
She said: “Harry’s a happy child who is full of life and a bit of a joker.
“He’s only six-years-old but he needs to use a wheelchair as the muscles in his legs are so weak.
“The anxiety about lack of funding for research and the pace of the search into treatments for Harry’s condition is a real issue for us.
“However, what is even worse is knowing that a treatment may be there for our son in the future, but that there will be barriers at every turn to Harry receiving it.
“We need the process speeding up – clinical trials, licensing and funding for medication. Our son and other children do not have the luxury of time.”
Mrs Vaughan is hopeful the inquiry will make people sit up and take notice.
“When Harry needs specialised treatments, these treatments may not be available, because of the restrictions that we may face.
"Harry has a limited life span and he may not get the same chance as other children or teenagers.
“I’m hoping that people, the public and politicians, will listen.
“At the end of the day, these are our children and if we can get a good stand behind us, more can be done.”