New cystic fibrosis medication means the future is much brighter for children such as Annabel Murphy.
Annabel was tested for CF as part of the newborn bloodspot programme that was introduced here in 2011.
She was just three weeks old when she was diagnosed with the disease — but being identified early has improved her quality of life.
Newborn screening means that children with CF can be treated with a high energy diet to improve weight gain and physiotherapy and medicines to improve lung function and gut absorption.
But it is largely because of advances in medical research that children like Annabel are thriving and living full lives.
She was one of the first children in Ireland with CF to be prescribed Orkambi in April 2018 and, fortunately, was able to tolerate it.
Thankfully, the future is bright for children like Annabel because of medical research on new CF medication.
This week it was announced that Irish researchers from the Royal College of Surgeons in Ireland will lead a €3m study on new CF medication.
The study, starting in August, will look at how a new triple combination CF drug, Kaftrio, will affect people in their day-to-day lives, rather than in controlled clinical trial conditions.
Kaftrio (known as TriKafta in the US) is a successor to Orkambi and Sarah hopes that Annabel will be prescribed the drug soon.
The drug has already demonstrated striking improvements in clinical trials of people with the most common form of CF and there is an agreement in place to fund the drug for people in Ireland.
“Annabel is doing really well at the moment but it would be great for her to get a drug that has such amazing health effects,” says Sarah.
“A lot of people with CF who are taking the drug at the moment have experienced a significant increase in lung function. Medical research is building brighter futures for children like Annabel who can take her medication and get on and enjoy her life just like any other eight-year-old.”
The two-year research project, called RECOVER, has been awarded €2.85m from Cystic Fibrosis Foundation (US), €112,000 from Cystic Fibrosis Trust (UK) and €100,000 from Cystic Fibrosis Ireland.
In partnership with the Irish and British cystic fibrosis registries, RECOVER is led by an Irish research team collaborating with researchers in 16 clinical and university sites in Britain, Europe and North America.
Chief executive of Cystic Fibrosis Ireland, Philip Watt, says researchers will look at the impact of new and innovative CF medications across Europe and the US.
As well as collecting routine health measures, they will also examine detailed imaging, functional, biological and quality of life measurements that could not be collected in clinical trials.