Irish child with killer brain disease OK'd for New York gene therapy

Irish community in New York raise $120,000 to pave the way for her recovery

In excess of $120,000 was raised and will contribute towards the cost of Saoirse’s trial.

Committee members and friends selling raffle tickets in their local boroughs raised over half the money. Thousands of dollars came through the door on Sunday in a fun filled family day of music and entertainment.

An auction of signed sports memorabilia and vacations to Ireland raised more than $10,000, and several private donations from the Irish community also contributed to the pot and overall success of the day.

Saoirse’s grandparents, Mike and Mary Carey, flew in from Ireland for the event and were overwhelmed with the success.
Billy O’Sullivan, co-chairperson of the event, said the committee was “overwhelmed with the support and generosity that people showed leading up to and on the day of the fundraiser.”

O’Sullivan, from Kenmare, Co. Kerry added, “We are very much appreciative to all the volunteers, the bands who gave up their time, the entertainers, the Kerrymen’s Association for the use of the hall and everyone that helped out to make this such a huge success.”
On the amount raised, O’Sullivan said, “We are very, very happy and money is still coming in.”

“Not only was it a fantastic collaboration between Kerry and Cork, it was great to see so many people from every other counties do their bit especially in the economy we are in but the Irish always pull through,” he added.

Said Tony, “We are delighted and so grateful to all our friends, old and new in New York, who have been and continue to be a great support to us all during this time.”

Saoirse and her two-year-old brother Liam suffer from Batten Disease, a regressive disorder of the brain that normally develops in childhood. Early symptoms of the disorder usually appear between the ages four through 10, with gradual onset of vision problems, or seizures.

Saoirse was diagnosed when she was four, while Liam at just one and a half.

Over time, affected children suffer mental impairment, worsening seizures and progressive loss of sight, speech and motor skills.

Doctors in Ireland told the Heffernan family that Saoirse and Liam would eventually die from the disease.  Not happy with the prognoses, Tony and Mary went in search of alternative treatments and found their answer at Cornell Hospital.

Saoirse’s surgery on October 12 is the first phase of an ongoing 5-year treatment plan.  The surgery will initially involve injecting a harmless gene-bearing virus into her brain. It has been found in a previous trial to significantly slow the progression of the disease.

The Stage II trial is aimed at extending the life expectancy of those with the disease, and providing greater quality of life.  It is also an important step towards finding a cure.

The Heffernans have their flights booked and are in the process of organizing accommodations near Cornell so they can be close to their daughter when she begins the treatment.

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Nster.com