Scientists, clinicians and patients will gather at the University of South Florida’s Center for Advanced Medical Learning and Simulation (CAMLS) in downtown Tampa Sept. 6 to share research insights and energize the search for a cure for Friedreich’s ataxia and related disorders.
The fourth annual scientific symposium “Understanding a Cure” will be held from 6 to 8 p.m. on Thursday, Sept. 6, at USF Health CAMLS, 124 South Franklin Street, Tampa, FL 33602. The symposium, free and open to the public, is hosted by Friedreich Ataxia Research Alliance (FARA) and the USF Ataxia Research Center (ARC), and will include a tour of CAMLS.
This year, for the first time, the scientific symposium will be live streamed through the FARA Facebook page, with opportunities for visitors to join the discussion long distance. To connect to the live stream and learn about cutting edge research advances in Friedreich’s ataxia, visit: http://www.facebook.com/CureFA/app_196506863720166 on September 6 at 6 p.m. A Facebook account is not needed to join.
Friedreich’s ataxia is a rare, debilitating neuromuscular disorder. Symptoms, typically emerging between ages 5 and 15, often progress to severe disability and include the following: loss of coordination and muscle weakness that leads to wheelchair use, energy deprivation and fatigue, vision impairment, hearing loss, slurred speech, aggressive scoliosis, diabetes, and life-shortening cardiac disease. There is not yet an approved treatment or a cure.
“This year’s Friedreich’s Ataxia Symposium will build upon the momentum of the last three years by highlighting some of the most promising discoveries yet from leading researchers in the field,” said Theresa Zesiewicz, MD, professor of neurology and director of the University of South Florida Ataxia Research Center, who moderates the symposium. “The opportunity for patients who cannot be with us in person to join the interactive symposium in real-time via Facebook has generated a lot of excitement. We’re looking forward to lively discussions.”
Symposium speakers will include Helene Puccio, PhD, research director for INSERM, Department of Translational Medicine and Neurogenetics, Institute of Genetics and Molecular and Cellular Biology (IMCBG) in Illkirch, France. Dr. Puccio’s laboratory develops animal and cell models to help understand the pathophysiology of Friedreich’s ataxia and the function of frataxin, a protein severely deficient in people with Friedreich’s ataxia. The preclinical models are used by Puccio and other researchers worldwide searching for potential new treatment approaches to ataxias, including gene replacement therapy.
Pediatric cardiologist R. Mark Payne, MD, professor of pediatrics and medical and molecular genetics director at Wells Center for Pediatric Research-Indiana University School of Medicine, will share his expertise on cardiomyopathy, or deterioration of heart muscle, related to Friedreich’s ataxia. Dr. Payne conducts research on heart disease caused by mitochondrial defects.
Guy Miller, MD, PhD, CEO of Edison Pharmaceuticals, Inc., will speak about advances in developing drugs to treat mitochondrial diseases that share a common feature – defects in how the body makes and regulates energy metabolism.
FARA President Ron Bartek and Jennifer Farmer, FARA executive director, will address progress nationwide in the research and management of Friedreich’s ataxia.
Stephen Klasko, MD, CEO for USF Health and dean of the Morsani College of Medicine, will moderate a panel discussion on patients’ perspectives of living with ataxias.
USF is one of 11 sites included in FARA’s Collaborative Clinical Research Network, an international network of centers that share data and resources to advance treatments and clinical research for people with Friedreich’s ataxia.