July 21, 2016 ~ Clearwater Beach, FL

Pathomechanism(s) of Barth Syndrome

Recapitulating the Barth cardiomyopathy in the inducible mouse model of tafazzin deficiency
Colin K. L. Phoon, MD, MPhil — New York University School of Medicine, New York, NY

The growing family of disorders in the biosynthesis and remodelling of phospholipids; the brothers and sisters of Barth syndrome
Saskia Wortmann-Hagemann, MD, PhD — Head, Department for Metabolic Disorders, Children’s Hospital Salzburger Landeskliniken; Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria

Elevated hepatic fatty acid oxidation contributes to low body weight in tafazzin knock-down mice
Laura Cole, PhD — Children’s Hospital Research Institute of Manitoba, Winnipeg, Manitoba, Canada

KEYNOTE SPEAKER

What we as families, advocates, scientists and industry partners can do to move Barth syndrome more fully into the clinical treatment era
Ronald J. Bartek, Co-Founder/Founding President, Friedreich's Ataxia Research Alliance (FARA)