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Expert Series: Understanding Upcoming MCADD Trials

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October 26 @ 12:00 pm 1:00 pm EDT

There are two new MCADD trials that have started in recent months. One explores the use Dojolvi (triheptanoin) and the second the use of phenylbutyrate with MCADD patients. Join MitoAction as Dr. Arnold discusses the science behind both studies, why these substances are being evaluated, and what these studies will look like. If you have any questions about the trials or about participating in either trial, we encourage you to come, learn, and bring your questions!

About the Speaker

Dr. Georgianne Arnold

Dr. Georgianne Arnold

Dr. Arnold graduated from Indiana University with degrees in Biology and Chemistry. She has a Master’s Degree in Medical Genetics from Indiana University-Purdue University at Indianapolis and graduated from medical school at Upstate Medical Center in New York. She completed a residency in Pediatrics at Northwestern University and a fellowship on Genetics at the University of Colorado. Dr. Arnold is board certified in Pediatrics, Clinical Genetics and Clinical Biochemical Genetics.

Previously, Dr. Arnold was faculty at the University of Arkansas and the University of Rochester. She is now Professor of Pediatrics and Clinical Research Director at the University of Pittsburgh Medical Center. Her main interest is clinical research in inborn errors of metabolism. She is currently conducting numerous clinical trials for a variety of metabolic disorders and is the Longitudinal Project Principal Investigator for the national Rare Disease Consortium for phenylketonuria.

Dr. Arnold has authored more than 70 medical publications, and received the Shapira Award for the best publication by a Society for Inherited Metabolic Disorders member in the journal Molecular Genetics and Metabolism for her paper “A Delphi clinical practice protocol for the management of very long-chain acyl-CoA dehydrogenase deficiency.” She has presented papers at numerous national and international meetings and is currently the President of the Society for Inherited Metabolic Disorders.