10-year-old Grace McIntyre's wish has come true - she can now, once again, avail of the life changing drug, Vimizim
The family of 10-year-old Aughavas school-girl, Grace McInytre, have expressed their elation after the HSE agreed to resume funding the life-changing medication, Vimizim.
Speaking to the Leitrim Observer this afternoon, Grace's mother Barbara said the whole family was "absolutely delighted".
"It was the best news we could get that the HSE had decided that Grace get get her medicine back. It is amazing. We are elated," she said.
Grace, who lives in south Leitrim, has the rare condition Morquio A Syndrome. People who have Morquio A can’t make an important enzyme that is a key part of clearing out materials from cells. This is a progressive condition and a buildup of materials in the cells can lead to potentially serious problems impacting the heart and skeleton as well as causing issues with vision, hearing and breathing.
It is an extremely rare condition and only 10 people have the syndrome in Ireland.
Grace and an 11-year-old Kilkenny girl, Cezy Fosca, are the only children with Morquio who were included in trials for Vimizim, an enzyme replacement therapy (ERT) which is the only treatment available for this condition.
Grace received weekly intravenous transfusions of the drug, Vimizim, since May 2012 as part of the trials. Her last treatment was on February 15 when her family were informed their were no more drugs available for Grace.
The drug was initially provided by the manufacturer, Biomarin, to Grace, free of charge on compassionate grounds. But it was withdrawn in February after a number of applications by Biomarin, to include the drug on the HSE reimbursement scheme, were refused. At the time, Biomarin indicated they could not continue to fund the drug indefinitely while waiting for inclusion on this scheme.
Sinn Féín Sligo/Leitrim Deputy, Martin Kenny, who has been working on behalf of Grace and Cezy Fosca to have their Vimizim supply reinstated, told the Leitrim Observer this afternoon that the administration of the drug has been approved through a managed access programme under contract with Temple Street Children's Hospital.
"Grace and Cezy will now receive the drug through a contract with Temple Street. The hospital will manage their access to their drug and monitor them on a continual basis. The great news is that the HSE is agreeing to fund their drugs going forward and that is a huge relief for the girls and their families," he said.
"I'm delighted with the outcome," he said. "The HSE Leadership group has accepted Temple Street's programme and the drugs will be restored and funded. That was always our goal."
But the real excitement will certainly be in the homes of Grace and Cezy and their families this evening.
"This is the best news ever," said Grace's mother. "I want to thank everyone who has helped us. All our friends and family, Deputy Martin Kenny, who worked so hard on this and also Cllr Paddy O'Rourke. Grace's school in Aughavas who have bent over backwards for her and everyone who have sent us so many messages of support. They have all been so wonderful.
"Today's news, is a dream come true."