Limavady mum Laura Smith will find out on Monday if a two-year battle to secure a life-changing new drug for her terminally ill son has been successful.
Her son, Callum, has a condition known as Duchenne Muscular Dystrophy which means he is unlikely to live beyond his twenties without access to a new drug known as Translarna.
Laura has been campaigning, along with other parents of children with Duchenne across the UK, for Translarna to be made available.
On Monday, April 18, the body who decide whether new medicines can be prescribed on the NHS will publish its guidance on Translarna. The body, known as the National Institute for Clinical Excellence (NICE), will decide whether it will be available in England and Wales with Northern Ireland set to follow suit.
Laura said: “In between all his appointments physio and medication, Callum is a typical wee boy. He loves Lego, minecraft and dinosaurs. He is thoughtful and kind, funny and loving, grumpy and stubborn. We will get a life changing decision. He knows nothing of it. He is so innocent and carefree. My wish is for this innocence to continue. I’m trying so hard to be positive about him getting his drug, because the alternative is too much to bear.”
The Limavady mother has already had an application to the Health and Social Care Board for the drug to be made available to Callum turned down.
Now, approval from NICE is Laura’s last remaining hope for her son. Translarna, also known as Ataluren, will not cure his condition but is likely to alleviate his suffering and should extend his lifespan significantly.
However, if Callum does not get access to it before he loses the ability to walk, it will be too late. He already requires a wheelchair from time to time but is still eligible for Translarna, for now.
Laura explained: “The NICE guidance applies across the UK but it is still more or less the NHS England. Northern Ireland will be different, the Isle of Man will be different and Scotland will be different. Dean Sullivan, the man in charge of commissioning drugs for the Health and Social Care Board in Northern Ireland, has assured us that what NHS England does, Northern Ireland will absolutely follow suit. That’s all we know for now until we get the guidance from NICE.”
Charity Muscular Dystrophy UK said: “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.”