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 and Events
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Super Bowl
RaffleIts time for our annual Super Bowl Raffle for GSD! Purchase a raffle ticket and you could be going to California for Super Bowl LX on February 8th! -
Beam
Clinical TrialsBeam Therapeutics is continuing to enroll adult participants in a Phase 1 / 2 trial evaluating BEAM-301 for the potential treatment of GSD1a. There are three clincial study sites open across the United States. Please contact clinicalinfo@beamtx.com for more information.
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Ultragenyx submits
BLA to FDA!Last week, Ultragenyx announced the initiation of a rolling submission of a BLA to the U.S. FDA seeking approval for their investigational gene therapy for patients with GSD1a. They plan to complete the BLA submission in the fourth quarter of 2025. Click the graphic to learn about the process and the timeline.
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GSD1a Meeting with the FDA
On October 9, 2025 The Children's Fund for GSD Research hosted an Externally Led Patient Focused Drug Development (EL-PFDD) Meeting where patients and caregivers shared their stories to help shape the future of treatment for GSD1a. Please visit the EL-PFDD information page to learn more and watch the Live Stream recording. -
Gene Editing
Clinical TrialsHuman clinical trials have started and the first GSD1a patient was dosed using novel gene editing technology. We are so grateful for the GSD teams at The University of Connecticut, UConn Health and Beam Therapeutics for getting us here.
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Research at UConn Continues
We are thrilled to continue our support of Dr. Youngmok Lee and the GSD Lab at UConn Health. Thank you Dr. Lee for your dedication and commitment to find a cure for Glycogen Storage Disease.
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" ... because every child deserves to be healthy."