New drug would lengthen and enhance my son's life. All we need is Edwin Poots' approval
Published 08/08/2014 | 02:30
A Northern Ireland family hope that a revolutionary new drug could transform the life of their son who suffers from a rare muscle-wasting condition.
For the first time a treatment for Duchenne muscular dystrophy is available and comes in the form of a drug called Translarna, which has just been approved by the European Commission
If given to children before they are unable to walk, it prolongs their mobility and enhances their short lives.
One child affected is little Callum McCorriston from Londonderry.
His mother Laura has battled to get the drug for him but fears the high costs may affect the Health Minister's decision to fund Translarna, even though Callum (5) is one of only two boys in Northern Ireland who are suitable.
Laura welcomed the EC approval as a step in the right direction but said it was frustrating that even though it could be available in England, her son may still lose out because he lives here.
Callum is in the race of his life to get the drug because right now he fits the criteria perfectly, but if he loses his ability to walk he will be denied Translarna.
For now Callum remains oblivious to his plight, plays happily in his garden and is just looking forward to starting primary school in his home town of Limavady in a couple of weeks' time.
He is a charismatic, content wee boy who puts up with the physiotherapy he has to do every morning and evening to keep his muscles flexible while his anxious mother awaits a decision from Edwin Poots on whether he will release the funds that will allow Callum access to Translarna.
Speaking to the Belfast Telegraph, Laura said: "Up until a few 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 getting the approval from the European Commission now is wonderful.
"But it is so frustrating that despite this, Callum might still not get it if the Health Minister thinks it is too costly to fund.
"There are only two boys in Northern Ireland who have Duchenne and are still on their feet, which means they are suitable for Translarna, so it is not as if Mr Poots will have to fund the costs of treatment for hundreds of children.
"Callum is due to start primary school soon, but without this drug, by the time he reaches P7, he will be in a wheelchair and by the time he is ready to leave secondary school, he won't be able to lift a glass to his lips, go to the bathroom for himself or reach over and hug us good night.
"Then the Duchenne will affect his chest and heart muscles... and after that, death.
"We know that this drug won't cure Callum but it will delay the onset of all of that by years and could extend his life from his early 20s to well into his 30s.
"It will also enhance the quality of the life he has – and what parent wouldn't fight for that for their child?"
A spokeswoman for the Department of Health said: "This licensing decision is conditional on the manufacturer completing its clinical trials process and submitting additional data regarding safety and efficacy. When this work has been completed, then the drug will be further appraised for its clinical and cost effectiveness. When new therapies are assessed and gain approval from NICE, the department seeks to ensure their introduction as soon as is practically possible."