JOIN A PATIENT REGISTRY
Patient registries for rare diseases are a fundamental research effort which support 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.
IamGSD is engaged with two registries – CoRDS and Euromac, for people affected by McArdle disease and other rare neuromuscular glycogenoses where exercise intolerance is the main symptom. (That is all muscle GSDs except GSD2 Pompe, which has its own registry).
Coordination of Rare Diseases at Sanford
IamGSD has worked with Sanford Research to create a registry specific to muscle GSD within their CoRDS international patient registry for all rare diseases.
Our IamGSD questionnaire is primarily aimed at patients in North America, but is open to patients worldwide. The registry is only available in English. Patients may self-register on-line, or request a paper document.
Euromac is a initiative of the European Union and was launched in 2013. It now has around 400 patients registered. Data is entered by medical professionals who have been appointed to the task.
Although open to patients worldwide, in practice due to the professional entry approach, it is almost exclusively patients attending European centres for muscle GSDs that have been entered. The Euromac web site and publications are available in nine languages.
The data has already been used to report on these conditions and to source patients for multi-centre trials.