International Clinical Outcome Study for Dysferlinopathy (LGMD2B/Miyoshi)
UPDATE: Click here to read the first participant newsletter.
An opportunity for patients with dysferlinopathies to participate in a clinical research study. This study is essential for finding outcomes that can be measured during clinical trials. Without good clinical outcomes, effective clinical trials cannot be performed.
Please click here for a list of the 14 worldwide study locations.
The Jain Foundation along with Dr. Kate Bushby at Newcastle University is coordinating the study across the 14 centers. The study is funded by the Jain Foundation. Additional details, including contact information, can be found in the "Who is running the study" question in the FAQ section.
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.
Please visit the pages below for more information about the International Clinical Outcome Study:
What is the goal of the study?
The clinical outcome study will evaluate 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.
Who is eligible?
All ambulant and non-ambulant patients age 10 and older with a genetically confirmed dysferlinopathy diagnosis are eligible for the study.
What information is collected?
Medical, physiotheraphy and MRI/MRS assessments will be 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.