ON THE ROAD TO A CURE

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

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. 
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!

News & Events

  • Super Bowl
    Raffle!

    And the winner of this year’s Super Bowl raffle is David O’Brien from Westlake Village, CA. This year we sold 790 raffle tickets and including additional donations raised over $100,000 dollars!! Thank you to everyone who participated!
  • December 2023 Newsletter

    Catch up on the latest GSD news and research from The Children's Fund. View our digital December 2023 Hopes and Dreams Newsletter below.  
  • Catching up with Jerrod Watts

    We recently sat down with Jerrod, the first GSD1a patient to receive Gene Therapy.  Here is how he is doing today.  
  • GSD1a Leadership Council

    Ultragenyx Pharmaceuticals is establishing a Global Leadership Council to learn more about the needs and challenges of the GSD1a community.  If you would like to be considered, please click below to learn more.  

  • Patient
    Spotlight

    Running a marathon is not an easy task for anyone. Running a marathon with GSD is something else entirely. With hard work, determination, and lots of smarties, this past November, Jake Gordon completed the NYC Marathon. Congrats Jake!

  • Gene Therapy Ph1/2 Trial Participants

    If you participated in the DTX401 Ph1/2 Clinical Trial, Ultragenyx invites you to participate in a confidential virtual interview. You will be compensated for your time.  If interested, please click on the image above for more information.
  • mRNA Trials
    Underway!

    The first patient in the world received Moderna mRNA trial infusion for GSD1a at UConn Health.
  • Investigational
    mRNA Treatment

    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.  

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

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