Matthis Synofzik, MD
University of Tübingen
Professor Dr. Matthis Synofzik, MD, is head of the research unit “Translational Genomics of neurodegenerative diseases” and senior consultant neurologist at the Hertie Institute for Clinical Brain Research & Center of Neurology, University of Tübingen, Germany. He leads the outpatient clinics for Frontotemporal Dementias and for Amyotrophic Lateral Sclerosis, and is co-chair of the Ataxia outpatient clinics at the Center for Neurology. Using latest next-generation genomic sequencing techniques, he has been leading or involved in the identification of >15 novel ataxia and spastic paraplegia (HSP) genes in the last 8 years. Matthis leads several worldwide EU-funded consortia on rare neurological diseases like PREPARE, PROSPAX” and EVIDENCE-RND. He is Vice Chair of the global trial-readiness platform “ATAXIA GLOBAL INITIATIVE”- a worldwide multicenter translational initiative to pave the way for all crucial translational steps from NGS genetic fingerprinting to FDA-conform trial-readiness cohorts of ataxia patients. His work in the N1C perfectly extends this large-scale endeavour and can directly draw on these global patient, biomaterial and NGS datasets and expertise for individualized n-of-1 ASO treatments. As part of the steering committee of the 1 MUTATION 1 MEDICINE (1M1M) consortium, he has already charted the path to establish this exciting novel therapy approach of ultra-individualized ASO treatments in Europe (Synofzik et al, 2021, Nucleic Acid Therapy).