What are the goals of the study?
The clinical outcome study is evaluating a large group of dysferlinopathy patients to determine which tests are best at measuring changes in the disease. The goals are to:
Define the best tests for measuring disease progression for use in clinical trials.
Collect biological samples for the identification of disease markers that are needed to non-invasively monitor the disease during clinical trials.
Collect accurate details about the full clinical spectrum of the different forms of dysferlinopathy by measuring disease progression. This information will help enhance patient care.
The outcome measures identified in this study are needed for the development and success of future clinical trials. In addition, the information gained in this study will provide a better understanding of the disease, ensure optimal care and treatment, and will lead to new and better therapies.