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Scientific, Medical & Family Conference

Family Sessions

July 19, 2018 - Clearwater Beach, FL

Potential Therapies for Barth Syndrome

Gene therapy vector optimization and testing for Barth syndrome
Christina Pacak, PhD, University of Florida, Gainesville, FL

Elamipretide (MTP-131) in subjects with genetically confirmed Barth syndrome (TAZPOWER): A phase 2 randomized, doubleblind, placebo-controlled crossover trial followed by an open-label treatment extension
Hilary J. Vernon, MD, PhD, Johns Hopkins University, Baltimore, MD
(36:58-01 - 01:14:08)

Evaluating antioxidant therapies in a tafazzin-knockdown mouse model of Barth syndrome
Colin Phoon, MPhil, MD, New York University School of Medicine, New York, NY
(01:14:08 - 01:53:19)

Identification of novel mitochondrial targeting peptides in tafazzin and long-term efficacy of enzyme replacement therapy in a mouse model of Barth syndrome
Michael Chin, MD, PhD, Tufts Medical Center, Boston, MA
(01:53:20 - 02:28:37)

Cross-species omics integration identifies new potential treatments for Barth syndrome
Riekelt Houtkooper, PhD, Academic Medical Center, Amsterdam, The Netherlands
(02:28:37 - 02:54:23)

Can elamipretide, the first cardiolipin-protective compound, benefit Barth syndrome patients?
Hazel Szeto, MD, PhD, Social Profit Network, Menlo Park, CA
(02:54:23 - 03:31:15)

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