Ira A. and Mary Lou Fulton Chair in Motor Neuron Diseases
Director, Gregory W. Fulton ALS and Neuromuscular Disorders Center, Barrow Neurological Institute
Associate Professor of Neurology, University of Arizona
240 W. Thomas Rd.
Phoenix, AZ 85013
Why did you enroll? I enrolled in the study because they were looking for study participants, with very specific criteria. I know that there aren't very many people with my type of dystrophy, in the general population, and I wanted to help future Miyoshi patents, if not myself to test out future therapies. It's not a lot of fun, but, without patients to study, they can only model different theories without any data to find out for sure. I hope my small part, can help, with their research for a cure.
Why did you enroll? I chose to take part in Dr. Zimmerberg’s research for the “greater good” and an unanticipated benefit is I’m learning an amazing amount of detail about my overall health, LGMD, and how the disease is affecting me.
Hello, my name is Kathy and I have LGMD and I’m sure if you are reading this you most likely have some form of Muscular Dystrophy (MD) and know that you are not alone.
Why did you enroll? I feel like it is so important to know as much as I can about this disease, for myself and for others. I feel lucky to be involved.
Myostatin is a protein in muscle cells that controls muscle growth. Reducing myostatin causes the growth of pre-existing muscle cells, enlarging the muscle fibers. When myostatin is reduced in healthy animals, muscles grow significantly larger.
As a prerequisite for future human clinical trials of treatments for dysferlinopathy, it is necessary to better dysferlinopathies in the absence of treatment. This will provide a baseline for future studies, as well as assist in the clinical design of future trials. In particular, it will help define how many patients will be needed to participate in trials, what duration of the trial will be necessary to observe an effect of treatment, and which clinical evaluations are most informative as to a patient's condition.