OUR RESEARCH AGENDA
We want to contribute to the success of research projects which deliver tangible advances that are highly relevant to patient needs.
IamGSD is committed to sharing the patient voice, perspective and experience in the following ways.
CONTRIBUTING THE PATIENT VOICE
IamGSD is well placed to gather and collate the needs and opinions of muscle GSD patients and their families. The planning of the research agenda has many factors influencing it – funding, staffing, reliability, etc. It is important not to lose sight of patient needs. IamGSD can bring the patient voice to the professionals involved with research so that the opinions of the patient group are a major factor in focussing the research effort.
PROMOTING PATIENT REGISTRIES
IamGSD works with two patient registries – CoRDS and Euromac. We encourage all people with a muscle GSD to register with one of them. Patient registries for rare diseases are a fundamental research effort which supports a number of activities. They are key instruments for increasing knowledge by pooling data on patients for fundamental and clinical research, epidemiological research, and observational studies.
We will encourage research where it aligns with what we believe are the best interests of muscle GSD patients. This can include contributing to research proposals, collating information from patients through surveys and workshops, supporting applications and in some circumstances may include assisting with funding.
Muscle GSDs are ultra rare conditions and as such it can be extremely difficult to recruit sufficient numbers of patients for trials. We can assist with recruitment through our IamGSD registered users, through our Facebook groups and other social media, and working with our partner national associations. We can raise awareness amongst the muscle GSD community and encourage involvement in patient registries and clinical trials.
When results are published, IamGSD will help to promulgate the news throughout the patient community. We will financially support Open Access wherever possible. Where results impact on the management advice given to patients, we will look to make this clear through our web site and to update our publications as necessary. We will endeavor to translate the technicalities of research findings into practical guidance for patients.