Background
A FOUR-year-old boy with a rare form of muscular dystrophy could be denied life-enhancing drugs because he lives in Northern Ireland.
Little Callum McCorriston was diagnosed with an unusual strain of MD — Duchenne — when he was two.
The muscle-wasting condition means it is unlikely he will live beyond his 20s.
Callum enjoys digging in the garden and playing on his toy tractor, but by the time he is ready to leave primary school he will probably be in a wheelchair.
A breakthrough drug called Translarna, which could keep the little Limavady boy mobile for years, will be offered to children with his condition living in England.
But because he lives in Northern Ireland there is no guarantee that he will get it.
Callum's mother Laura is taking her fight to get her son the drug to Stormont on Monday, where she will lobby the Health Minister.
Callum remains oblivious and is just looking forward to starting primary school in September.
Laura said: “When Callum was born we had no idea anything was wrong and it wasn't until a Duchenne muscular dystrophy is a neuromuscular condition caused by the lack of a protein called dystrophin. It causes progressive muscle weakness. About 100 boys with Duchenne muscular dystrophy are born in the United Kingdom each year.
Guidelines which allow children with Duchenne in England to access the drug Translarna are not used here, so at the moment no one knows if or when it will be introduced in NI. health visitor was making a routine check-up when he was 15 months that she noticed he wasn't reaching some of the expected targets.
“Tests were done and we got the devastating news a couple of weeks later. Up until two weeks ago there was nothing that could be done to save our son, and then we heard about this new drug.
“All along we hoped and prayed that there would be some kind of breakthrough, and this is it.
“Right now Callum fits the criteria that will allow him to get the drug — he will be five in July and he is still on his feet.
“He is one of just two boys in Northern Ireland that are suitable, but my overwhelming fear is that by the time Translarna is available here Callum will be in a wheelchair and unsuitable.
“That's what makes this so urgent. We do not have time on our side because, sadly, Callum could walk upstairs one night and not be able to walk down again the following morning, that is how uncertain his future is.”
A spokeswoman for the Department of Health said the treatment of rare diseases is a high priority for the minister, Edwin Poots.