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.
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What have we learned so far?
Preliminary results from the study
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What you can do to make the study better.
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, a necessary step for the development and success of future clinical trials.
What information is being collected?
Medical, physiotheraphy and MRI/MRS assessments are being performed on 6 occasions over 3 years.
The study will be conducted in 14 centers across Europe, USA, Japan, and Australia. The study is free for all participants and funds are available for patients and a helper to travel to the nearest study center.
Frequently Asked Questions
Learn more about the International Clinical Outcome Study by reading some of the most frequently asked questions.