Limavady mum ‘beyond delighted’ at Translarna drug news


A Limavady mum says she is “beyond delighted” at a decision to recommend funding for a drug which she believes will help her son.

Muscular Dystrophy UK says the National Institute for Health and Care Excellence (NICE) has recommended funding on the NHS in England for Translarna - “the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy”.

“There is now a legally required consultation period on the implementation of this positive guidance before the assessment process is formally completed on May 25,” the charity says. “It is fantastic news that we are close to boys being able to access Translarna on the NHS in England. The priority now is to make sure that NHS England implements the decision as soon as possible.”

The charity says it expects NICE’s decision to lead to implementation in Northern Ireland, Wales, and the Isle of Man.

“Today we are writing to Health Ministers in Northern Ireland, Wales and the Isle of Man to ensure that there is no delay to accessing Translarna,” said a spokesperson for the charity.

Limavady woman Laura Smith says she hopes the decision is implemented soon and that Northern Ireland will follow suit.

Her son Callum, aged six, has the devastating muscle-wasting condition Duchenne Muscular Dystrophy and Laura, along with Muscular Dystrophy UK and hundreds of other parents across the UK, has been fighting for Translarna to be made available in Northern Ireland.

Laura says the NICE decision is “amazing”.

“I am in total shock,” she told the ‘Journal’. “The decision wasn’t due until Monday so this news today is totally unexpected. What it means for Callum, well, as far as I am aware, it will be available in the UK so we shouldn’t have an issue with him getting it in Northern Ireland.”

Nice has said patients who are eligible will be able to have access for five years.

In those five years, the children will be monitored to check their progress, and then the drug will be reviewed.

Laura says Translarna would be life-enhancing for Callum.

“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.

“We have been fighting for this for so long and, having been turned down for funding for it for Callum three times, I was really starting to believe we were fighting a losing battle. It is amazing news and I am beyond delighted. It’s just amazing, but it will take a while to sink in.”

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