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.
News & Events
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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 on the image above to learn more.
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Patient
SpotlightRunning 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. Please join us in congratulating him on this amazing accomplishment!
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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 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. -
2022
GSD GalaWe had a blast at "A Night in Nashville" at the Seminole Hard Rock Hotel & Casino in Hollywood, FL. Thank you to all of our sponsors, supporters and friends for making it such an incredible event.
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" ... because every child deserves to be healthy."