Health Minister Edwin Poots’ approval for a new drug is now all that is needed to change the life of a little boy from Limavady.
Little Callum McCorriston, a four-year-old boy from Limavady, suffers from a rare and acute form of muscular dystrophy, known as Duchenne.
Without treatment, the condition will cause a steady deterioration in his muscles, leaving him wheelchair bound by the time he attends secondary school and paralysed by his late teens. He is not expected to live beyond his twenties.
Researchers had failed to come up with any effective treatment - until now. A new drug, known as Translarna, can be used to help sufferers whose condition is caused by a specific genetic mutation and little Callum is amongst that small group.
In fact, he may be the only boy in Northern Ireland with the condition who can be helped by the new drug.
His mother, Laura Smith, has been campaigning for better care for some time and has expressed fear that the drug may not be made available on the NHS because he lives in Northern Ireland - rather than in England.
In fact, Health Minister Edwin Poots recently wrote to Laura to explain the process whereby the drug could be made available. Mr Poots said: “In Northern Ireland, arrangements are in place through routine commissioning to ensure appropriate and timely evidence-based access to medicines, including specialist drugs like Translarna.
“The Health and Social Care Board and the health authorities in England are guided by the same appraisal organisation, the National Institute for Health and Care Excellence (NICE), when considering the introduction of new drug therapies. When new therapies are assessed and gain approval from NICE, the Department seeks to ensure introduction as soon as is practically possible.”
However, the drug has now gained the approval of the National Institute for Health and Care Excellence, so it is now essentially up to the Minister whether the drug can be made available in time to change the life of little Callum from Limavady.
Laura said: “Edwin Poots basically told me in his letter that he was waiting for approval from the NICE organisation. That has now been granted so it is really down to him now. You have to ask the Minister whether he thinks my son is worth spending the money for the Translarna. If it is a question of money for the drug at this stage you have to ask the Minister if my son’s life is worth it.”