What we are funding now

Our ultimate goal is to live in a world without Glycogen Storage Disease. 

Your generous contributions are working hard to help us get there.


Investigational mRNA Treatment

Research is underway on an investigational mRNA treatment to see if it can correct the cause of GSD1a by teaching the body to break down glycogen.  Click below to learn more about the Ba1ance Trial.  


Gene Editing

Beam Therapeutics has initiated a clinical development program in glycogen storage disease type 1a.  Beam is using base editing to potentiall correct the causative genetic change in G6PC: initially the R83C variant, which is the most common mutation in GSD1a.  Base editing is an emerging class of precision genetic medicines designed to overcome the limitations of existing approaches and expand the potential of genetic medicine.  By rewriting a single base, base editors may correct disease-causing point mutations and potentially create life-long cures for patients suffering from serious diseases. Click below to read about their preclinical data.

Gene Therapy

Ultragenyx Pharmaceuticals is evaluating DTX401 to establish normal glucose metabolism and reduce or eliminate the need for cornstarch to maintain normal glucose levels.  The company is currently in phase 3 of clinical trials.  




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" ... because every child deserves to be healthy."

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