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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FDA Approves Treatment for GSD1a!
On August 19, 2026, the U.S. FDA approved the first-ever treatment designed to address the underlying cause of Glycogen Storage Disease Type Ia. This marks a huge milestone for the GSD community. Please talk to your doctor to find out if gene therapy is right for you. Learn more about Genglycos by clicking below. -
EL-PFDD Meeting
with the FDAOn October 9, 2025, The Children's Fund for GSD Research hosted an Externally Led Patient Focused Drug Development Meeting (EL-PFDD). This meeting gave patients and families an opportunity to share their voices and stories with the purpose of accelerating research and improving care. -
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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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."