The International Clinical Outcome Study for Dysferlinopathy

The Clinical Outcome Study for Dysferlinopathy is an opportunity for patients with dysferlinopathy to participate in a clinical research study, essential for defining outcome measures necessary to assess the effectiveness of potential therapies during clinical trials.

We hope to be able to recruit at least 150 participants worldwide. Recruitment opened in Autumn 2012.

Oversight and funding of this study is provided by the Jain Foundation. This study is jointly led by Professor Kate Bushby of Newcastle University and Dr. Laura Rufibach of the Jain Foundation and is conducted in 14 centres worldwide.

Please note that participants DO NOT need to live in the study centre cities to participate. Funds to cover travel costs to the study centres for the participants and a helper, if necessary, are available.

Members of the International Dysferlinopathy Registry will receive updates regarding the Clinical Outcome Study for Dysferlinopathy.



What is a clinical outcome study? 

Who is eligible? 

What is the purpose of this study? 

Why should I participate in this study? 

What specific tests and measurements are included in the study? 

Where can I get more information?