Almost 22 years ago The Children’s Fund for GSD Research, Inc. was created, and thanks to you, we have raised over $8,000,000 dollars! Most of these funds have been granted to researchers around the world, all working to make the lives of people with GSD1 and their families better, with the hopes of one day ultimately curing this disease. We are proud to have helped fund preclinical animal studies which were the gateway towards moving into clinical human trials! 

Although we are pleased with the progress and outlook so far, we have not reached our goals yet. We still worry about the health of our children every second of every day and for this reason, we will continue to work hard raising funds so that we can support the world’s most promising GSD1 research. We won't stop until a cure is found.

Ultragenyx completed a Phase 1/2 human clinical study and is currently more than halfway through the fully enrolled Phase 3 trial. “The Phase 3 GlucoGene study is underway to evaluate the ability of DTX401 (the company’s moniker for the gene therapy ‘drug’) to reduce the use of cornstarch while maintaining or improving glucose control as well as the therapy’s impact on patients’ quality of life.” This is huge! Your generosity has and continues to make a difference. We are happy to share the exciting data from the Phase 1/2 Study as well as the press release from UConn Health about the Phase 3 trials.

Moderna has been researching the use of mRNA for the potential treatment of GSD1a. The Balance Moderna Trial, which began at UConn Health in June 2022, aims to see if a new investigational medication, mRNA-3745, is safe and if it could potentially help correct the defect that causes GSD1a and restore function. If successful, mRNA-3745 would teach certain cells in the body to effectively break down glycogen, correct low blood glucose and avoid starch intake. To learn more, read the Uconn Health press release about the first patient dosed.

Moderna Trials

Beam Therapeutics, located in Cambridge, Mass., is dedicated to providing lifelong cures to patients suffering from serious diseases. Beam's technologies, which are based on gene editing, seek to address unmet medical needs so that people can reach their full potential. Beam-301 is focused on Glycogen Storage Disease Type 1a and is currently in its IND enabling stage. It is focused on gene correction via a LNP in vivo modality. This study is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, which is the most common mutation responsible for causing GSD1a. 

To learn more about this exciting advancement in GSD research, please read Beam's October 25, 2023 press release addressing the status of new preclinical data demonstrating the ability of its drug candidate to directly correct the R83C mutation.

Beam Trials

The GSD laboratory at the University of Connecticut (UCONN) has been created for Dr. Youngmok Lee where he and his team work towards developing cures through preclinical research using animal GSD models. The lab raises awareness about GSD and gene therapy among research communities, collaborating with research groups around the world. At UCONN there are both ongoing and future projects related to GSD1a. Read about them HERE.

Recently, board member Teri Reed sat down with Jerrod Watts, the first GSD1a gene therapy patient as he shared his inspirational experience being patient 1 of phase 1 human trials, which began July 24, 2018….

What made you decide to participate in the GSD1a gene therapy trial as patient 1?

When I was a child, my goal in life was to bring about a change in the treatment of GSD so no one in the community would ever again fear dying in the middle of the night due to a missed cornstarch dose. 

How has your life changed since your treatment?

My life has been a complete 180 since the trial. Not only is my glucose under much better control, but I have also seen improvements in my body on a molecular level. 

Did the results meet your expectations?

Actually, I went into the trial with no expectations for my own treatment; my only hope was that it would not make me sick. 

Read the full interview HERE

On November 5, 2022, The Children's Fund for GSD Research hosted our 13th fundraising gala, A Night in Nashville. Over 300 friends, family members, sponsors, scientists, researchers and colleagues gathered together at the Seminole Hard Rock Hotel & Casino in Hollywood, Florida to support one another and learn about the progress we have made in GSD Research. Read more here.

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Running a marathon is not an easy task for anyone. Running a marathon with GSD is something else entirely. When my son Jake first began to exercise in middle school, his blood sugar would constantly drop to dangerously low levels. Making it just one lap around the school track was a difficult task, with rescue measures put in place to bring his blood sugar level back to stable numbers. But Jake was determined to make this work. Through hard work, determination, and communication with his care team, he turned himself into an accomplished runner. Read the full story.