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MELAS Spectrum Disorders and Sonlicromanol: Progress Update

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Join MitoAction and Dr. Jan Smeitink, CEO of Khondrion for our our March Expert Series presentation. MELAS spectrum (m.3243A>G) disorders belong to the most frequently encountered group of primary mitochondrial diseases with an unmet medical need for treatment development. Following an introduction of the clinical disease spectrum Dr. Smeitink will discuss the development and state-of-art of Khondrion’s lead product sonlicromanol.

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About the Speaker

Jan Smeitink, MD, PhD

Jan Smeitink, MD, PhD

Jan Smeitink obtained his training in Pediatrics at the Radboud university medical center, Nijmegen, The Netherlands whereafter he completed a 3 year training in metabolic disease at the Wilhelmina Children’s Hospital, Utrecht, The Netherlands.  From 1996 until May 2020 he was the Head of the Department of Metabolic Diseases at the Radboud university medical center with a special interest in the study of mitochondria in health and disease. Since May 2020 he is fully employed as Chief Executive Officer of Khondrion BV.  

Smeitink has more than 25 years of experience in patient care, diagnostics, counselling and research in the field of primary mitochondrial disease. In 1996 he  founded the Radboud Centre for Mitochondrial Medicine. He has initiated, supervised and collaborated in many national and international research programs, and has published over 400 peer reviewed scientific articles (including N Eng J Med, Science, Nature Genetics, Cell Metabolism, American Journal of Human Genetics, Lancet Neurology: H-index above 80). He has been invited to give more than 100 lectures including many key-lectures, and seminars all over the world and has organized key meetings and courses on mitochondrial medicine that significantly contributed to the continuing success of the field (Euromit conferences, Frontiers in Bioenergetics, Systems Biology of Bioenergetics, focused courses on Mitochondrial Medicine).

 Smeitink received the Prinses Beatrix Foundation Jubilee Award (2006) for his research on mitochondrial medicine and an Honorary Membership of the Pediatric Neurology association of Hong Kong (2011). He has given numerous interviews to national and international newspapers, radio stations and television. From 2008-2014 he was Director of the Institute of Genetic and Metabolic Disease one of the leading research institutes of the Radboud university medical center. From 2010 - 2015 he was chairman of the Centre for Systems Biology and Bioenergetics.  Prof. Dr. Smeitink has obtained several major grants and was the coordinator of the 6th Framework Program EUMITOCOMBAT (2004-2008) funded by the European Commission and the ongoing KHON2TREAT 7th Framework Program Grant. Four of his former students have become full professor at international institutes, while most others have enrolled in research staff positions. In October 2013 he was awarded a membership of the Academia Europaea. In April 2016 he was honoured with the highly prestigious Knight in the Order of the Dutch Lion in view of his services to medical research. In September 2019 he was honoured with the RCMM Achievement Award in view of his contributions to mitochondrial medicine. Jan Smeitink has extensive collaborations with many mitochondrial patient organisations and researchers from all over the world.

Jan is the founding CEO of the clinical stage, pharmaceutical company, Khondrion (www.khondrion.com), which became fully operational in November 2012.  The company’s lead product sonlicromanol is currently in phase 2b and long-term open label extension studies in adults with MELAS spectrum disorders. Recently, the first children were enrolled in a phase 2 trial. 

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