MitoAction
Support, Education, Outreach and Advocacy for Children and Adults Living with Mitochondrial Disease
Expert Series: Hope on the Horizon: The Vital Role of Patients in Clinical Research
February 7 @ 12:00 pm – 1:00 pm EST
There is unprecedented momentum in the mitochondrial disease clinical trial landscape, and the patient community plays a vital role in ensuring these trials have the potential to lead to new and effective treatments. This expert series aims to demystify clinical trial participation and answer your most pressing questions. Dr. Amy Goldstein, Clinical Director of the Mitochondrial Medicine Frontier Program, and Chad Glasser, Sr. Director of Clinical Research at Tisento Therapeutics, will discuss the PRIZM MELAS study, what to expect if you participate in trials, and highlight the importance of clinical trials in the drug approval process.
About the Speaker
Amy Goldstein, MD
I am a child neurologist with a subspecialty focus and expertise in primary mitochondrial disorders. I have been the Clinical Director of the Mitochondrial Medicine Frontier Program since July 2017. Under my leadership, we have expanded the clinical service to include inpatient consultations at CHOP as well as the Hospital of the University of Pennsylvania (for adults), and we have quadrupled the outpatient patient volume. I am actively involved in direct patient care, clinical diagnosis, and management, striving to make ongoing improvements in patient care. We perform retrospective and prospective natural history studies and interventional clinical trials where I serve as Physician Lead.
In addition to my clinical service, my major contributions to the field of Mitochondrial Medicine include education and awareness. I serve the patients and families by volunteering my service for the major patients’ advocacy support groups. I am a past member of the Scientific and Medical Advisory Board for the United Mitochondrial Disease Foundation (UMDF) and have served as a medical advisor for MitoAction. I have served the clinicians who care for patients with mitochondrial disease as a board member and immediate past president of the Mitochondrial Medicine Society, and I am a founding member of the Mitochondrial Care Network. I am a contributing author to numerous publications and am frequently invited to give talks at national and international meetings. I am also a faculty member of CMEM (Center for Mitochondrial Epigenomic Medicine) and Professor of Clinical Pediatrics at the University of Pennsylvania, where I also hold a secondary appointment in Neurology.
