Cure GSD

ON THE ROAD TO A CURE

"... because every child deserves to be healthy."

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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. 

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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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Did you know…

The Children’s Fund for GSD Research is the only public charity in the world dedicated to funding research for Glycogen Storage Disease Type 1.
Over $7.5 Million Funded
Since 2002, The Children's Fund for GSD Research has granted over $7.5 million, which has led to groundbreaking therapies and an improved quality of life for people with GSD1. To date, we have funded over 55 studies, helping scientists pursue new ideas and investigate probable approaches to improve treatment and uncover a cure. 
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100% Volunteer Based
Our ultimate goal is a to live in a world where GSD does not exist. The Children's Fund for GSD Research is leading the charge towards this reality. All fundraising and administration are done on a volunteer basis so that nearly 100% of all contributions can be used to support our mission. Join our cause!
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News & Events

  • GSD Odyssey

    GSD Odyssey, a new study launched by Ultragenyx, enables someone with GSD to contribute their de-identified medical information to advance research. 
    GSD Odyssey Flyer

  • Gene Therapy

    What is gene therapy and is it right for you?
    Gene Therapy Brochure

  • Thank You!

    We are so grateful for everyone who participated in our 2020 Gift from the Heart fundraising campaign. If you have not yet donated, please consider sending us a gift so that we can keep this important research moving forward and change the lives of people living with GSD. Thank you!
    Donate Now

  • Gene Therapy
    Update

    We reached a milestone when the first dose of Gene Therapy was delivered to a GSD patient.  Preliminary results have been extremely positive. 

    Learn More

  • Community Update
    Webinar

    Thank 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. 

    Ultragenyx Webinar

  • 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.  
    Learn How
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" ... because every child deserves to be healthy."

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