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.
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
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Seeking GSD1a (R83C) Patients
RTI Health Solutions is hosting a series of interviews for GSD1a patients with the R83C mutation. The information obtained may be used by a pharmaceutical company developing a new treatment for GSD1a. Interviews are approximately 1 hour and participants will be paid $100 for their time. If interested, please click 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 TreatmentResearch 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.
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Gene Therapy
UpdateUltragenyx Therapeutics is now in Phase III clinical trials for GSD1a. The first participant recieved the infusion at Uconn Health in January 2022. -
Ultragenyx
Clinical TrialsResearch 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. -
GSD Odyssey
StudyGSD Odyssey, a patient study launched by Ultragenyx, enables someone with GSD to contribute their de-identified medical information to advance research.
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" ... because every child deserves to be healthy."