THE parents of a ten-year-old boy who suffers from a progressive muscle-wasting disease are hoping the drug that dramatically improved his condition will be made available on the NHS.

The National Institute for Health and Care Excellence met on Tuesday to review whether Translarna should be funded for the treatment of Duchenne muscular dystrophy, with a decision expected before Christmas.

Ruth and Damien Le Gal from Ivybridge say that if Translarna had been available and their son Leo had begun taking it earlier, he would still be able to walk – something the couple say the youngster is aware of.

Leo does currently get the drug, as he is taking part in a trial that sees him travelling to Great Ormond Street in London every twelve weeks.

Prior to this, the family had been making regular trips to The USA in order to take part in the trial.

Ruth said: ‘In the immediate future this won’t make any difference to him. It’s kind of alright for us, but on the other hand we’ve seen what a big difference it’s made to Leo not getting it early enough.

‘He’s only 10, but he knows that if he had started taking the medicine early enough, he’d probably still be able to walk.

‘That’s quite hard for a ten-year-old to have to deal with.’

In August 2014, Translarna became the first treatment for Duchenne muscular dystrophy to be approved in the EU.

While the drug was available in countries including Germany, France, Spain and Italy within months, families across the UK have faced an agonising wait for news on funding.

Children must still be walking to be eligible for Translarna, and with most needing a wheelchair before the age of 12, there are fears delays will mean some will miss out.

But last month NICE said it would not recommend NHS access to Translarna without more justification of its cost and evidence of its impact.

Ruth continued: ‘It’s devastating for people to know that there are really good drugs, that could save their kids, but they can’t get them.

‘It’s actually worse, I’d say, than the drugs not existing at all.

‘Sometimes the world is just a bit of a mad place.’

Chief executive of charity Muscular Dystrophy UK, Robert Meadowcroft, criticised the ‘convoluted’ process of getting drugs approved.

He said: ‘Families have hoped for decades for a way to intervene in the progress of Duchenne muscular dystrophy. Translarna is the first treatment to offer this chance.

‘We urge NICE to do all within its power to find a workable solution for delivering on this long-awaited breakthrough. The childhoods of many eligible young people across the UK could be transformed.

‘It is deeply encouraging that today, more and more therapies for Duchenne muscular dystrophy are reaching clinical trials. As Duchenne is progressive, we cannot afford to go through this convoluted and painful process each and every time a new therapy is in sight.

‘The emotional cost for families simply cannot be justified.’