Coalisland Mum appealing to FDA to approve drug that could save her son at US conference

Claire O’Hanlon, who lost her brother and three uncles to Duchenne, also saw five year-old Luke diagnosed with the muscle wasting disorder in May 2012.

It will see him wheelchair bound during childhood, completely paralysed and dependent on ventilation by his mid-teens and gives him a life expectancy of just 19.

But on April 23, Claire hopes she can make a difference for him at a conference in Washington DC.

She told the Times she feels “for the first time ever there is real hope” to improve Luke’s quality of life as well as that of her little cousin’s, Brian Og McStravog, who has the same condition.

Claire said: “I am travelling to the US to support the Duchenne community in putting pressure on the FDA to approve a drug called Eteplirsen.”

Eteplirsen helps patch over the missing DNA code (exon) that affects protein production, and has already helped the 12 children involved in trials walk well into their teens.

As it is a personalised drug, Claire said Luke and Brian Og would not be suitable, but passing it would pave the way for “follow on exon skipping drugs in trial which will help Luke and Brian Og”.

“These drugs will not cure the condition - but will be the first drugs approved that will help to change DMD from a terminal condition into a chronic and manageable one,” she added.