CAMPAIGN 6
CONTRIBUTING TO RESEARCH
THE ISSUE
Clinical and epidemiological data on muscle GSDs is limited. With only a few clinicians worldwide seeing significant numbers of patients, research opportunities are limited. There is a distinct requirement for establishing networks of patients and their families on an international basis, and ensuring that patients’ needs are fed into the discussions on the research agenda.
We support all types of clinical, lab and desk research.
OUR GOAL
To contribute to the planning of research projects and to support and assist those projects.
OUR OBJECTIVES
We will work towards our goal by:
Collaborating with researchers on translational research projects.
Promoting patient involvement in patient registries (Euromac and CoRDS).
Participating in committees that are responsible for research decision-making.
Disseminating information about research to patients and family members.
Providing funding to researchers.
OUR PROGRESS
We have established good relations with all the leading researchers worldwide, and frequently collaborate on translational research projects.
We promote both the Euromac Registry and theCoRDS registry on our website. When appropriate we mention them on social media.
We have not been invited onto bodies responsible for research decision-making, but do have close relationships with many researchers.
Our funds are very limited compared to research costs, but where we have been able to help is in funding Open Access fees of papers resulting from research.
THE FUTURE
This is, of course, an ongoing task until such time as maybe one day we get either a highly effective treatment, or a cure. Given the fast pace of development on gene therapy techniques, it may well be that this will eventually provide a cure for McArdle’s, but it is some years away yet. For the even more rare muscle GSDs it is likely to be many more years again.