The family of a Limavady boy with a rare muscle wasting disease are praying they are closer to a decision about whether their son will have access to a potentially life-changing drug.
Callum McCorriston suffers from Duchenne Muscular Dystrophy, a condition that causes gradual but inevitable muscular deterioration and eventually leads to paralysis and death.
A new treatment - Translarna - is the first ever drug to target an underlying genetic cause of a form of muscular dystrophy.
Callum, who turns seven today, is one of just a few children in the North who could benefit from the treatment, to target an underlying genetic cause of a form of muscular dystrophy.
Callum’s mum, Laura Smith, said they hope the Termoncanice P.S. pupil would get the drug following indications from the National Institute for Health and Care Excellence (NICE) - the body which decides whether new medicines should be available on the NHS - that Translarna should receive approval.
Now, a final agreement has been reached between NHS England and PTC Therapeutics on funding for Duchenne muscular dystrophy drug, Translarna, Laura has fresh hope the drug will become available in Northern Ireland this year.
“For now, it’s a matter of waiting, yet again.
“Seven is usually the age where you see a decline with Duchenne, where Callum might start falling down or losing his mobility,” said Laura, “so we can’t afford to waste anymore time.”
Callum will celebrate his birthday with his parents, Laura and Paul, family and friends.
Laura says the best birthday present would be news Translarna will be made available to boys with Duchenne in Northern Ireland.
“Callum turns seven today,” said Laura.
“We’re excited, and Callum is excited, but it’s bittersweet, and it’s hard for myself and Paul because we’re thinking ‘how many more birthdays will he have?’. I know it sounds drastic, but that’s our reality and that’s the message we have to get across to health officials.”
Laura said, hopefully, once the drug is available in England, she will meet health minister, Michelle O’Neill.
The charity, Muscular Dystrophy UK, which has been campaigning for the drug, has said: “The fight goes on for equality of access in Scotland where we have urged the First Minister to act decisively and urgently to end the current ‘postcode lottery’. In Wales and Northern Ireland, there is a clear understanding they will follow NICE guidance when it is published.”