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).