Advancing research and improving lives

Prior to 2002, there was very little information available about Glycogen Storage Disease and research was virtually non existent.

Since then, The Children’s Fund for GSD Research has had a major impact on the GSD community.


By raising awareness and funding research, we are improving the lives of patients and families and advancing research towards a cure.  Below we outline our main initiatives to generate significant impact in our GSD community.

Provide Financial Support

The Children's Fund has granted over $8M to support cutting edge research.


To date, we have supported 62 studies to help scientists pursue new ideas and investigate probable approaches to improve treatment.


Spread Awareness

The number of children that have been properly diagnosed has nearly tripled thanks to increased awareness and knowledge made possilbe through literature, programs, and events.


The Glycogen Storage Disease program at the University of Connecticut, which is partially funded by The Children's Fund, now cares for over 600 patients from 49 states and 51 countries.


Glycogen Storage Disease has been added to the prenatal screening panel for genetic diseases.


Education has almost completely wiped GSD out of the Orthodox Jewish population, previously the group with the highest prevalence.

Support Novel Research

The Children's Fund helped to advance Gene Therapy by supporting various studies focused on development, safety and efficacy.


Commencement of Phase 1/2 Gene Therapy trials in GSD1a in July 2018.


Commencement of Phase 3 Gene Therapy trials in GSD1a in January 2022.

Improve Lives

The Children's Fund helped to discover and obtain FDA approval for the use of "superstarch" by GSD patients, allowing them to safely sleep through the night. Glycosade was approved in 2012. This was the first major breakthrough in the dietary management of GSD in over 25 years.


Continously providing education and support to build a stronger GSD community.


Actively funding studies to prevent long-term health related issues


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

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