A MUM from Hythe is lobbying the Government to push through a bill which will allow her terminally ill son to access promising medicine that could prolong his life.

Claire-Rose Young is calling for more to be done to help her seven-year-old son Charlie who was born with Duchenne Muscular Dystrophy – a fatal genetic disorder that affects one in every 3,500 males.

His condition means Charlie probably won’t live past 20.

It also means simple tasks such as eating and walking independently are something he will not be able to do in the future. He has no dystrophin in his body, meaning muscle cells are easily damaged, which can lead to serious medical problems particularly with his heart and lung.

Now Miss Young has launched A Chance for Charlie – a fundraising campaign which aims not only to help her son but also other children in her fight to get access to new medicine.

Lord Saatchi is campaigning to change the law that, with consent, will allow patients to access drugs being trialled – some of which can take up to 10 years to receive formal sanction – commonly known as the ‘right to try’ bill.

At the moment the only recognised treatment is through steroids which can have serious side effects.

But the tide may be turning for these little boys following the announcement that an American pharmaceutical company is meeting with the US Food and Drug Administration with a pre-new drug application (NDA) regarding their product eteplirsen.

In a statement released Dr Edward Kaye, Sarepta Therapeutic’s interim chief executive officer and chief medical officer, said: “We will initiate a rolling NDA submission to facilitate the regulatory review of the NDA.

“The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible.”

But it remains to be seen if the new drug, even if it is successful in the US, will ever be rolled out in the UK.

Miss Young, of Hollybank Road, had even written to Nick Clegg – when he was deputy prime minister – begging him to consider “right to try” drugs – linked to the recent Saatchi bill – to at least give Charlie a fighting chance. “There are many trials out there but not many are getting good results,” she said. “The one in America is very promising and all boys on trial have shown real improvements, some now out of wheelchairs, feeding themselves again.

“The US FDA keep stipulating they need more data to approve the drug. This can take years boys don’t have. And the trouble is there are not enough boys to trial as this drug has only been designed for boys with certain deletions,” she added.

“We emailed Nick Clegg asking for the right to try and he said that it was such a complex issue and needed looking into further before discussing it in Parliament - wasting more time boys don't have. All we wanted was it discussed in Commons. Our dream would be for the FDA to pass Sarepta’s drug so that the EU has to look at bringing it here. That drug would work for Charlie and I would love him on it. Unfortunately it’s not being accelerated and looks a long way off and even then the NHS here would have to fund it.

“Our dream is to find a cure and give Charlie a chance of a life that every child should have.”

Miss Young, 27, has held a number of mini events to raise money and awareness of Charlie’s condition, including a recent craft event at Poppies Café in Romsey, which raised £279.

But Charlie needs so much more if he’s got a chance at being more independent. While his bungalow is a nice size with a warm feel, strict NHS guidelines means his home needs alterations to ensure he’s as comfortable as possible.

“We want to give him as much independent living at home so it doesn’t feel like a hospital space,” Miss Young said.

He enjoys his hydrotherapy pool at Naomi House where the family also get counselling when required, and it’s a place where Charlie can have a fun break away from hospital visits.

For more information or to donate to Charlie visit achanceforcharlie.com.