International Clinical Outcome Study for Dysferlinopathy (LGMD2B/Miyoshi)

Over 200 individuals are participating in the International Clinical Outcome Study (COS), well exceeding our initial goal of 150 patients. Every participant is critical because it is such a rare disease.  Therefore, we need for all participants to complete all 6 visits of the study so that we have enough data to do the necessary analysis and draw conclusions from the data.

The Jain Foundation along with Dr. Kate Bushby at Newcastle University is coordinating the study across 15 centers worldwide. The study is funded by the Jain Foundation.

Why is this study so important?

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.