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.
Duke University, Durham, N. Carolina, USA has been involved in research for GSD3. The link to some of their work is below.
They also have a Facebook page for their GSD3 Clinical and Research Program.