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Cori disease (GSD3)

Research & Development

GSD3 is rare yet there are researchers, scientists and doctors carrying out studies with the intention of improving the quality of life for those affected.

The research agenda

The AGSD-UK likes to promote research by consulting with proposals, facilitating meetings and providing funding where possible.

Patients can help by help volunteering to participate in any ongoing studies and being part of the consultation process.

Developing gene therapy

Gene therapy is a possible treatment for GSD3. Ultragenyx has reported that their mRNA program, UX053, for the treatment of GSD3, is progressing well and AGSD-UK understands this to indicate that a clinical trial in GSD3 may start before the end of 2020.

Go to the Ultragenyx web site.

Go to our news story on the Ultragenyx tie up with Arcturus.


Duke University

Duke University, Durham, N. Carolina, USA has been involved in research for GSD3. The link to some of their work is below.

 Link to Duke University research

They also have a Facebook page for their GSD3 Clinical and Research Program.

 Link to Facebook page