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Hundreds to attend Florida fundraiser for rare Ashkenazi disease

(JTA) – Some 300 people are expected to attend a South Florida fundraiser for research on treating GSD, a rare Ashkenazi Jewish liver disorder. 

Glycogen storage disease, which blocks the liver’s ability to convert glycogen into glucose, affects fewer than 200,000 individuals worldwide and is so rare in the United States that it does not receive federal funding, Lisa Hodes, vice president of the Children’s Fund for Glycogen Disease Research and fundraising chair, told the Florida Jewish Journal.

Donors will convene for dinner at the Coral Springs Marriott on April 19, she said. Seats cost $150. A golf tournament at $275 per player will be held before the dinner.

Families with members suffering from the disease rely on philanthropy to fund research, Hodes, whose two teenage daughters suffer from the disease, told the Jewish Journal. 

GSD patients must maintain a strict diet and eat every one to four hours.

"We work with gene therapy, and the goal of this therapy is to give the patients a copy of the gene that does not have a mutation in it," Thomas Conlon, an assistant scientist of pediatrics at the Gene Therapy Center, told the Jewish Journal. "We have cured this disease model in dogs. We need additional funding in the millions to bring this to phase-1 human trials."

With enough funding, clinical trials could begin as early as spring 2014, according to the report in the Jewish Journal. It also said the disease was recently added to the Ashkenazi Jewish disease testing panel.

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