July 21, 2016 ~ Clearwater Beach, FL

Potential Therapies for Barth Syndrome

Update on exercise and substrate metabolism studies in Barth syndrome
W. Todd Cade, PT, PhD — Associate Professor of Physical Therapy and Internal Medicine, Washington University School of Medicine, St. Louis, MO; Scientific and Medical Advisory Board, Barth Syndrome Foundation

Tafazzin enzyme replacement therapy for Barth syndrome
Michael T. Chin, MD, PhD, FACC, FAHA — Associate Professor of Medicine, Harold T. Dodge-John L. Locke Chair in Cardiovascular Medicine, University of Washington, Seattle, WA

Targeting PPAR/PGC1alpha axis in Barth syndrome: The PPAR pan-agonist bezafibrate ameliorates cardiomyopathy in a mouse model of Barth syndrome
Zaza Khuchua, PhD — Associate Professor, The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Assessing the effects of Bezafibrate on the cardiomyopathy induced by adult-onset knockdown of tafazzin in a mouse model
Mindong Ren, PhD — New York University School of Medicine, New York, NY

Update on pre-clinical studies to develop gene therapy for Barth syndrome
Christina A. Pacak, PhD — University of Florida College of Medicine, Gainesville, FL

Clinical, molecular, and metabolomic studies in Barth syndrome: Outcomes from the 2014 Barth Syndrome Foundation Scientific, Medical & Family Conference
Brittany DeCroes Hornby PT, DPT, PCS — Kennedy Krieger Institute, Baltimore, MD