Cure GSD

ON THE ROAD TO A CURE

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

Gene Therapy Update

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 $8 Million Funded
Since 2002, The Children's Fund for GSD Research has granted over $8 million, which has led to groundbreaking therapies and an improved quality of life for people with GSD1. To date, we have funded almost 60 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

  • Gene Therapy
    Update

    Ultragenyx Therapeutics is now in Phase III clinical trials for GSD1a.  The first participant recieved the infusion at Uconn Health in January 2022.  
    Read the Press Release

  • mRNA Trials
    Underway!

    The first patient in the world received Moderna mRNA trial infusion for GSD1a at UConn Health.
    Read the Press Release

  • Ultragenyx
    Clinical Trials

    Research Opportunity:  Click on the photo above to learn how you can participate in the Phase 3 gene therapy clinical trials or the link below to learn more about the study.
    Gene Therapy Trials

  • Moderna
    Clinical Trials

    Research is underway on an investigational mRNA treatment that could potentially correct the cause of GSD1a by teaching the body to break down glycogen.  Click below to learn more about the Ba1ance Trial.  

    Moderna Trials

  • What is
    Gene Therapy?

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

  • 2022 GSD Gala
    Save the Date!

    We are excited to announce our 2022 GSD Gala, A Night in Nashville at the Seminole Hard Rock Hotel & Casino in Hollywood, FL.  
    Tickets & Sponsorships
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" ... because every child deserves to be healthy."

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