The Jain Foundation is delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (COS), exceeding the original goal of 150 patients. Professor Kate Bushby, MD, at Newcastle University and Laura Rufibach, PhD, at the Jain Foundation are co-leading this multi-year study, running in 14 centers worldwide. The study is projected to cost over $3M and is funded entirely by the Jain Foundation.
Dysferlinopathy, also called Limb Girdle Muscular Dystrophy type 2B (LGMD2B) or Miyoshi myopathy, is a rare genetic disease that causes progressive muscle weakness. "Because the normal disease progression has not yet been defined, the main goal of the Clinical Outcome Study is to measure disease progression in patients at all phases of the disease and to identify tests that can be used in clinical trials. This study is a critical step towards developing a therapy for this disease because it will make it possible to test the effectiveness of potential drugs and interventions," explains Dr. Rufibach.
"The recruitment of this large number of people affected by such a rare disease is a real indication of the dedication of the study teams and the enthusiasm of the patients to participate in research," said Professor Bushby. Dr. Plavi Mittal, President and CEO of the Jain Foundation, confirmed this saying, "Because we are testing as much as possible in this study, we are asking a lot from everyone, especially the patients. I am continually impressed by the commitment of the patients and staff at all of our 14 COS centers." The study centers are located in the United Kingdom, United States, Japan, Australia, Germany, France, Spain, and Italy. For more information about the study, visit www.jain-foundation.org/dysferlinoutcomestudy.