Cure GSD
WHAT IS GSD1?
Glycogen Storage Disease Type 1 (GSD1) is a rare, genetic metabolic disorder that occurs when a specific enzyme is either missing or not functioning properly. This enzyme is responsible for maintaining the body's blood glucose (sugar) level. Glucose fuels every cell in our body, including brain activity. People affected with GSD1 cannot convert stored glycogen into glucose, and therefore need a constant external source of glucose in order to survive. Hypoglycemia in patients with GSD1 can be life threatening. GSD1 is the most common form Glycogen Storage Disease, affecting 1 in every 100,000 births. READ MORE
The Children's Fund for Glycogen Storage Disease Research is a public not-for-profit 501(c)(3) foundation that aims to make a difference in the lives of children and their families affected by GSD1.
As little as 40 years ago, a child born with GSD1 had very little chance of survival beyond one or two years. Today, thanks to increased awareness, medical advances and generous supporters, children are thriving and a cure is on the horizon.
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News & Events
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GSD Odyssey
GSD Odyssey, a new study launched by Ultragenyx, enables someone with GSD to contribute their de-identified medical information to advance research. -
Share your Story!
FDA patient Affairs and NORD are hosting two "Listening Sessions" on GSDI. These small, private, and confidential conference calls will allow people affected by GSDI to share their experiences first-hand with the FDA. There are a limited number of openings. If you are interested in participating, please click the link below by February 16th to fill out the brief survey. -
Gene Therapy
UpdateWe reached a milestone when the first dose of Gene Therapy was delivered to a GSD patient. Preliminary results have been extremely positive.
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Community Update
WebinarThank you to Ultragenyx for providing updates on the investigational GSDIa and GSDIII programs. If you were unable to join live, you can view the recorded webinar by clicking below.
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Does your shopping give back?
You can help us fund promising GSD research with your online shopping at Amazon, iGive, and ebay, at no extra cost to you.
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The Children's Fund for
Glycogen Storage Disease Research
20 Sherwood Lane
Cheshire, CT 06410
203.272.CURE (2873)
" ... because every child deserves to be healthy."