Leigh Syndrome global patient registry
First results have been presented at Mitochondria-Targeted Drug Development Summit, February 22-24, 2022
Authors: Sophia Zilber, MA, Kasey Woleben, Danielle Boyce, DPA, MPH, Kevin Freiert, MBA, Joana Matos, PhD, Courtney Boggs, RN, Saima Kayani, MD, Souad Messahel, PhD
Abstract: Cure Mito Foundation started Leigh Syndrome (LS) Global Patient Registry in September, 2021 to meet the following goals: internationally available, identify and learn about LS patient population, consistently share results, enable clinical trial recruitment. Data collected during the first three months since starting the registry is high quality and describes LS population well in terms of demographic and disease related information. Participants represent 22 countries, over 70% international, achieving possibly the first global registry for LS. Future efforts include continued patient recruitment, publication of further results, collaboration with patients, industry, researchers.
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