2013:
2013
A Potential New DMD Therapeutic
Posted February 26, 2013
Gail Thomas, Ph.D., Cedars-Sinai Medical Center
Duchenne and Becker Muscular Dystrophy are X-lined muscle wasting diseases for which there are limited treatment options. Dr. Gail Thomas at Cedars-Sinai Medical Center hypothesizes that the nitric oxide donating drug naproxcinod may increase muscle blood flow in patients with muscular dystrophy thereby slowing disease progression. Dr. Thomas' research team will evaluate the effects of naproxcinod treatment on skeletal muscle and cardiac function in an animal model. The long-term goal of Dr. Thomas' research is to establish naproxcinod as a therapeutic to arrest muscular dystrophy disease progression and improve quality of life as well as to extend life.
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DMD Diagnostic Optical Imaging
Posted February 26, 2013
Glenn Walter, Ph.D., University of Florida
One of the major limitations of identifying and evaluating novel DMD therapeutic interventions is the lack of effective techniques to monitor muscle cell response. Dr. Glenn Walter and his research team at the University of Florida will evaluate the ability of contrast agent indocyanine green to detect muscle damage, muscle blood flow, and drug delivery using near infrared (NIR) imaging techniques. The long-term goal of Dr. Walter's research is to establish NIR imaging, a low-cost nonionizing imaging technology, as a tool for monitoring muscle permeability and therapeutic agent delivery along with providing clinically useful information for diagnostic and prognostic purposes in patients with neuromuscular diseases.
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New Clinical Outcome Measurements
Posted February 26, 2013
Avital Cnaan, Ph.D., Children's Research Institute at Children's National Medical Center
Although there is no cure for DMD, several new therapies are under development. Current clinical trial outcome measurements, such as muscle strength, are not, however, correlated well with patient function or quality of life. Dr. Avital Cnaan and her research team at Children's National Medical Center will build upon the existing DMD natural history study to identify clinical trial outcome measurements that can be utilized in the planning of DMD clinical trials. The long-term goal of Dr. Cnaan's research is the development of practical and easily administered outcome measures that are sensitive and responsive to changes produced by treatments in children and adults with muscular dystrophies or other neuromuscular diseases across the lifespan and stages of disease severity.
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GALGT2 Gene Therapy
Posted February 26, 2013
Paul Martin, Ph.D., Research Institute at Nationwide Children's Hospital
A promising therapeutic avenue for DMD is the use of gene therapy to overexpress GALGT2, an enzyme that alters skeletal muscle glycosylation to boost the expression of proteins that ameliorate disease. Recent studies suggest that overexpression of Galgt2 in skeletal muscle can inhibit DMD disease progression in several muscular dystrophy animal models. Therefore, Dr. Paul Martin at the Research Institute at Nationwide Children's Hospital has developed gene therapy vectors for human use to deliver the GALGT2 gene to skeletal and/or cardiac muscle. Dr. Martin's research team will complete dose response studies in two DMD animal models that are necessary to move this novel therapeutic intervention into a clinical trial. The long-term goal Dr. Martin's research is to develop a systemic treatment for all the muscles of a DMD patient.
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