Mother takes fight for medication to the Dáil
THE Kerry mother of a six-year-old girl with a rare genetic disorder said she is delighted following her participation in a presentation in Dáil Éireann as part of a delegation calling for HSE and Government approval of drug Kuvan.
Bernadette Gilroy’s daughter Maria was diagnosed with PKU just 10 days after birth. She was limited for life to a low-protein diet, an extremely important measure for PKU sufferers, who cannot break down a certain part of protein, exposing them to risk of severe intellectual disability and potential physical disability.
Mrs Gilroy – who lives in Mounthawk, Tralee, with Maria, husband Michael, and 10-year-old son Ethan – explained to The Kerryman, however, that studies show existing dietary therapy, while effective, does not necessarily prevent problems including bone-mass reduction, nutritional deficits, growth retardation, weight problems and neurocognitive deficits. She also explained that the drug Kuvan, which has not yet been approved in Ireland due to cost issues, can enhance metabolic control, reduce neurocognitive deficits and allow more flexibility with the challenging diet.
“Several studies show that many people struggle to stick consistently to the diet, particularly as they get older,” she said. “Maria’s diet is made up of three components: a synthetic protein drink that provides 90 per cent of her nutrition; a daily allowance of natural protein that’s contingent on weekly blood tests; and medicated foods predominantly made of starch.
“Incidence of PKU in Ireland is unusually high, and the drug itself is manufactured just over the border in Cork. The NCPE has twice decided not to recommend it on cost grounds, even though it has been approved in 19 countries in the last decade. This means Irish citizens with an Irish condition cannot access an Irish-manufactured drug.”
The next step in the campaign for PKU will be taken by Mrs Gilroy tonight as she appears on The Pat Kenny Show between 10pm and 11pm on TV3, which she hopes will be as well received as the presentation she was part of on Thursday last.
“Available research and information tells us that Kuvan’s cost-effectiveness could be stronger than suggested. Patients would need less of the medicated foods they currently take, and another study shows that PKU sufferers using half the recommended dosage enjoyed enhanced quality of life.
“The feedback we got from a great turnout of TDs and Senators last Thursday was very positive, and by all accounts our presentation had a big impact. Further information on this condition is available at www.pku.ie, or the PKU Association of Ireland Facebook page.”
Bernadette Gilroy will appear on The Pat Kenny Show to argue the need for Kuvan to treat her daughter’s PHU diagnosis