Huge Florida fundraiser for rare Ashkenazi illness
Research into treatments for Glycogen Storage Disease does not receive US federal funding and relies purely on philanthropy
Add ToI as a Preferred Source on GoogleJTA – Some 300 people are expected at a fundraiser near Miami for research on treating Glycogen Storage Disease, a rare Ashkenazi Jewish liver disorder.
The 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, according to Lisa Hodes, vice president of the Children’s Fund for Glycogen Disease Research and fundraising chair.
Donors will convene for dinner at the Coral Springs Marriott on April 19, she said. Seats cost $150. A Golf tournament priced 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 both suffer from the disease, told the South Florida Sun Sentinel.
GSD patients must maintain a strict diet and eat every 1-4 hours.
Thomas Conlon, Ph.D., assistant scientist of pediatrics at the Gene Therapy Center, described the research.
“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,” he said. “We have cured this disease model in dogs. We need additional funding in the millions to bring this to phase-1 human trials.”
If enough funds are raised, clinical trials could begin as early as spring 2014, according to the report in the Sun Sentinel. It also said the disease was recently added to the Ashkenazi Jewish disease testing panel.
The Times of Israel Community.