The parents of a Limavady boy with a rare muscle wasting disease say they are “immensely happy” their son looks set to receive a new drug that could prolong his life.
Callum McCorriston suffers from Duchenne Muscular Dystrophy, a condition that causes gradual but inevitable muscular deterioration and eventually leads to paralysis and death.
The seven year-old’s parents, Laura Smith and Paul McCorriston, have been campaigning for the last five years for Callum to have access to Translarna - the first ever drug to target an underlying genetic cause of a form of muscular dystrophy.
After approval in England for the drug, Laura said indications had been made it was only a matter of time that Northern Ireland would follow.
However, health minister Michelle O’Neill today announced eligible patients in Northern Ireland will receive innovative new drugs for the treatment of very rare conditions including Duchenne muscular dystrophy caused by a nonsense mutation, which is what Callum suffers from.
“The news was totally unexpected. We got a phone call this morning and Michelle O’Neill tweeted me as well about it. We are still in a state of shock and, I suppose, until we have the prescription in our hand we won’t believe it,” Laura told the ‘Journal’.
“It means it will extend Callum’s life and keep him on his feet for longer. It’s not a cure by any means, but it will improve his quality of life.”
Callum is a pupil at Termoncanice P.S. in Limavady. Laura and Paul say their brave son isn’t aware of his condition, other than knowing he gets tired a lot, and sometimes he has to use a wheelchair.
Laura added: “It’s just immense. It has been the last two years, really pushing for this. It can’t come quick enough. Please God, it’s a matter of weeks before we have the prescription in our hands.”
Callum’s dad, Paul said: “I suppose we are being a bit reserved with the jubilation because until it happens we won’t believe it, but it’s great news. We know it’s not a cure for Callum, but it’s about increasing his life expectancy. It’s just great news.”
We are still in a state of shock and, I suppose, until we have the prescription in our hand we won’t believe it.Laura Smith
Health Minister Michelle O’Neill said: “I am delighted to announce the availability of these drugs to allow patients with very rare conditions to receive pioneering new treatments. Whilst there are only a handful of people affected by these conditions, the new drugs have the potential to make a real impact on their quality of life.
“For the very first time, there are drugs available that could keep children with one form of muscular dystrophy out of a wheelchair for up to seven years; that could avoid end stage renal failure for people with atypical Haemolytic Uraemic Syndrome; or could relieve some of the symptoms of mucopolysaccharidosis type IVa.”