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

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As news trickled across the Atlantic that five-year-old Saoirse Heffernan had been accepted as a candidate for a gene therapy trial at New York’s Cornell Hospital that will prolong and potentially save her life, the Irish American community in New York banded together to raise more than $120,000 on Sunday to pave the way for her recovery.

Saoirse, who lives in Keel, Co. Kerry, spent a week in New York last May undergoing various procedures to assess her strength for a Stage II trial.

Although her tests were favorable, Saoirse was put on a waiting list until last week.  

Saoirse’s parents, Tony and Mary Heffernan, received the news on Friday, September 24 that their only daughter had been accepted on the trial that is to be conducted at Cornell Hospital in early October.

“We received the news on Friday, exactly 52 weeks after Saoirse was diagnosed with Batten Disease,” Tony Heffernan told the Irish Voice from Ireland on Monday.

It was Mary who took the call from Cornell at home in Co. Kerry. Tony was at an important meeting in Dublin when he was frantically interrupted with the good news.

Tony had left instructions not to be interrupted unless it was an emergency. This was a good emergency in Mary’s eyes so she made the call.

“We were just overjoyed when we found out that Saoirse had been accepted but we are also apprehensive,” said Tony.

Although accepted on the trial, Saoirse will have to undergo a verification process when she arrives in the U.S.

“The verification process is to make sure she still meets the criteria for the trials,” explained Tony.

“We are very hopeful, but we don’t know what has happened on the inside of her skull so that will be determined when we get there.”
If all goes well during the verification process, Saoirse will have surgery on Tuesday, October 12. She will be admitted into Cornell on October 8.

In an all out effort by members of the Irish American community in New York, specifically a small group of Cork and Kerry people, to raise as much money as possible to help Saoirse and other children with Batten Disease in Ireland, hundreds of people turned up and emptied their pockets at a fundraiser in the Kerry Hall in Yonkers on Sunday.

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Nster.com