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Find similar grantsResearch Grants (for Glycogen Storage Disease Type 1) is sponsored by The Children's Fund for Glycogen Storage Disease Research (Cure GSD). While specifically for Glycogen Storage Disease Type 1 (GSD1), this foundation funds research into a rare genetic metabolic disorder.
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"... because every child deserves to be healthy." 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. 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.
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. 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!
On 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.
View the Meeting Beam 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. 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. 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.
GSD1a EL-PFDD Human 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. 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. Apply for Grant This template controls the elements: FOOTER : Footer Title, Footer Descriptions CUSTOM MENU : Images and columns into header main menu submenu items * This message is only visible in administrative mode Glycogen Storage Disease Research Privacy - Terms - Refunds " ... because every child deserves to be healthy."
According to the current listing, eligibility includes: Scientists pursuing new ideas and investigating probable approaches to improve treatment and uncover a cure for GSD1. Confirm the full requirements in the official notice before applying.
The current listing shows not specified (over $8 million granted since 2002). Verify award ceilings, matching requirements, and allowable costs in the official notice.
Research Grants (for Glycogen Storage Disease Type 1) is funded by The Children's Fund for Glycogen Storage Disease Research (Cure GSD). Verify program details on the funder's official page before applying.
Start from the official opportunity page linked in this listing — it carries the sponsor's submission instructions.
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