• Project Dates: From September 2012 to August 2017
• Project Leader: Professor Kate Bushby/Professor Volker Straub
• Sponsors: Jain Foundation

The “Clinical Outcome Study for Dysferlinopathy” is anticipated to begin in mid-2012 in centres in Europe (UK, Spain, Germany, Italy and France), USA, Japan and Australia. The aim of this “Clinical Outcome Study” is to determine the clinical outcome measures required for future clinical trials, characterize the disease progression of dysferlinopathy and collect biological samples for the identification of disease markers that are needed to non-invasively monitor the disease during clinical trials. Without this information, effective clinical trials cannot be performed.

Staff Profile: Professor Volker Straub and Professor Kate Bushby.