Hope for families as CF drug deal agreed

by Sylvia Pownall
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TWO Malahide mums say they “now have hope” following a decision to make the drug Orkambi available to Cystic Fibrosis (CF) sufferers from next month.
The HSE and drug manufacturer Vertex have agreed a deal meaning Orkambi, as well as the drug Kalydeco, will be available from May 1.
Teacher Elmarie Tipper, whose six-year-old son TJ has CF, told The Gazette: “I can’t believe it – it’s just amazing. This is the first time we’ve had hope.
“It will make a huge difference to TJ’s life. He’s had a very difficult time but hopefully now we can scale back on some of his treatments.”
Elaine Nolan, whose 16-year-old daughter Ruth is a CF sufferer, said: “We’ve been campaigning for this for the last two and a half years. It will benefit her hugely.”
Mum-of-four Elaine paid tribute to Ruth’s pals in transition year at Santa Sabina secondary school in Sutton which mounted a campaign and collected thousands of signatures to support her.
She told The Gazette: “I’m still in shock at the news. Ruth is an ideal candidate for the drug – it will stop her CF from progressing. It’s not a cure, but it will treat the underlying problems.”
The Government u-turn – announced in the Dail late on Tuesday evening – followed a lengthy campaign by parents after Health Minister Simon Harris said the funding for the drug could not be found.
Cystic Fibrosis Ireland yesterday welcomed the news, paying tribute to all in the CF community who had campaigned so long to get the drug approved.
A spokesperson said: “The development is especially welcome, coming as it does during Cystic Fibrosis National Awareness Week, with 65 Roses Day taking place countrywide this Thursday.”
Elmarie now hopes Orkambi will offer little TJ a better quality of life after a long and painful road and endless rounds of IV injections, nebulisor treatments and physiotherapy.
In a heartbreaking Facebook post, she recently revealed: “Bedtime isn’t easy for TJ. At six years of age he lies awake processing his day, why IVs and treatments are so unfair and as his sister lies beside him sleeping soundly, he is always thinking ahead to the next round of IVS.”
But yesterday Elmarie said the future looked brighter, adding: “I’m still in shock, but it’s a good kind of shock.”

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