Belfast Telegraph

'This won't cure Callum's illness but it'll enhance his life beyond our dreams... we couldn't be happier'

Case study

By Donna Deeney

The news that a life-enhancing drug for children with a rare form of muscular dystrophy is to become available in Northern Ireland hit Laura Smith like a bolt from the blue.

Her son, Callum McCorriston (7), could now be given Translarna within a fortnight, ending the Limavady family's two-year battle for access to the medicine.

Health Minister Michelle O'Neill confirmed the drug, which has been described as a "real lifeline" for children like Callum, would be funded by the health service, despite its expense.

Speaking to the Belfast Telegraph, Laura said: "We knew the National Institute for Health and Care Excellence guidelines were due for publication, and I was ready for a day of lobbying the Health Minister and others to remind them how important it was to my son to have Translarna. So it really was like a bolt from the blue when I got the call to say the minister had already approved it.

"We could not be happier or more relieved. We know Translarna will not cure Callum, and we accept that nothing is going to cure him, but this drug will enhance his life beyond all our dreams. During trials, children who were on Translarna remained mobile until they were around 14, which is twice as long as expected.

"Callum is seven now, and time was of the essence for him, because if he had lost his ability to walk, he would not have been suitable for Translarna.

"Callum is a bright, enthusiastic wee boy who loves to play in the garden and loves school and playing with his friends. This drug will allow him to continue all of that for longer.

"This has been a long battle for us, but we are grateful for the support we received, including that from our local politicians, especially Alan Robinson and Maeve McLaughlin, who both fought so hard on our behalf.

"While this is certainly a day of celebration for us, we are mindful of the families living in Scotland and in the Isle of Man whose sons have been denied Translarna, but we will continue to fight for them."

Belfast Telegraph


From Belfast Telegraph