A Limavady mum campaigning for her son to access a life-enhancing drug is praying for the “right decision” so the treatment will be made available.
Callum McCorriston, who turns 7 in July, has the devastating muscle-wasting condition Duchenne muscular dystrophy.
Laura, along with the charity Muscular Dystrophy UK, and hundreds of other parents in the UK, has been fighting for the drug to be available in Northern Ireland.
She will know on Monday, April 18th, if the campaign has been successful as the National Institute for Clinical Excellence (NICE) will decide whether Translarna will be available in England and Wales, with Northern Ireland set to follow suit.
However, Laura said she isn’t optimistic after the Scottish Medicines Consortium this week rejected the use of the drug because “there was too much uncertainty about the overall clinical benefits it might bring in relation to its cost”.
“The decision in Scotland that the SMC will not approve NHS funding for Translarna is disgusting. How can they bring cost into it because it is, effectively, children’s lives that we are talking about,” Laura told the ‘Journal’.
“I think with the decision in Scotland, we aren’t going to get a ‘yes’ from NICE. We won’t know until Monday, but that’s my gut feelling.”
Laura’s previous applications for funding for the drug to be made available to Callum have been turned down by the Health and Social Care Board.
Approval from NICE for Translarna, also known as ataluren, is Callum’s last hope.
In the meantime, Laura said Callum is oblivious to his plight and battles on.
“He’s not too bad. It’s just the same ole same ole; he has lots of appointments and he goes where he needs to go.
“We’ve been in Belfast twice this year and he has physio in Derry. He does what he has to, although he does ask more questions now. He’ll ask about sore legs and we’ll tell him he gets sore legs, but that everyone is different.”
Laura said if Monday’s decision doesn’t go the right way, it is likely there would be an appeal, but that could take months which is time Callum and other children can’t afford.
“It’s time they don’t have and, I mean, how many more children will come off their feet by the time an appeal is dealt with?
“I am worried because Callum has put a lot of weight on because of the steroids, which puts extra strain on him,” said Laura. “Callum is still mobile, but I notice he is a lot stiffer. I genuinely don’t know which is worse - having a condition with no treatment, or having a condition with treatment you can’t access. It’s like the carrot is being dangled in front of you but you can never reach it, but I suppose we’ll know on Monday.”
Laura added: “I honestly don’t think unless you have a child with the condition you understand, or understand the significance of the drug.”
Muscular Dystrophy UK posted on social media: “We have received news from NICE that the decision on Translarna will be announced on Monday 18 April. NICE has also released a publication date of 25 May 2016 for its final assessment for the Duchenne muscular dystrophy treatment.”