Emeritus Professor Marc C. Patterson, MD, FRACP, FAAN, FANA Marc Patterson was born and educated in Australia, where he was educated, and graduated with first class honors in medicine from the University of Queensland in 1981. He trained in neurology and child neurology at the University of Queensland (1982-1988), in child neurology at Mayo Clinic (1988-1990) and neurometabolic disease at the Developmental and Metabolic Neurology Branch, NINDS/NIH, under the direction of Roscoe Brady, MD (1990-1992). Dr. Patterson is a fellow of the Royal Australasian College of Physicians and is certified by the ABPN in Neurology with Special Qualification in Child Neurology, and in Neurodevelopmental Disabilities. He served as Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic through his retirement in December 2024. During his tenure at Mayo, he was appointed as Director of the Child Neurology Training Program (2008-2016), and Chair of the Division of Child and Adolescent Neurology (2008-2017). Dr Patterson had previously served as Professor and Director of Pediatric Neurology and Director of the Child Neurology Training Program at Columbia University in New York (2001-2007). Following his retirement from practice, in January 2025, Dr Patterson joined IntraBio, Inc., as US Chief Medical Officer, continuing his focus on developing safe and effective therapies for neurodevelopmental and neurodegenerative disorders. Dr Patterson served as Editor for the Journal of Inherited Metabolic Disease and JIMD Reports, Editor-in Chief of the Journal of Child Neurology and Section Editor (Pediatric Neurology) for Up-To-Date through his retirement from practice. He has previously served on the editorial board of Neurology and the oversight committee for Annals of Neurology. Dr Patterson served as an examiner and committee member for the ABPN for more than 20 years and was Vice-Chair of the Neurodevelopmental Disabilities MOC committee through December 2024. He has previously served on the RITE examination committee of the American Academy of Neurology (AAN), as program director for educational courses at multiple AAN annual meetings, and as a counselor and scientific program director for the Child Neurology Society (CNS). His research and practice have focused on rare diseases in children, including inherited metabolic disorders in general, with special interests in Niemann-Pick disease, type C (NPC), other lysosomal diseases, and congenital disorders of glycosylation, areas in which he has published more than 250 peer-reviewed papers and book chapters. He led the pivotal trial which led to the approval of miglustat for the treatment of Niemann-Pick disease, type C in the European Union and more than countries worldwide; he has also participated in the design and execution of clinical studies which led to the recent FDA approval of arimoclomol and N-acetyl-L-leucine for the treatment of Niemann-Pick disease, type C, in the United States. Dr Patterson has served on the scientific advisory boards of several rare disease foundations and chaired the scientific advisory board for the international Niemann-Pick disease registry, an entity supported by a consortium of international Niemann-Pick Disease Foundations, and with an initial grant from the European Union. He has received funding support from NIH, industry, and private foundations.

Federico Goodsaid is Principal Consultant at Regulatory Pathfinders (since 2017), for regulatory strategy of therapeutics and diagnostics companies for precision medicine, digital, imaging, and AI products. He was previously Senior Vice President for Regulatory and Quality at Ariana Data Intelligence, a US subsidiary of Ariana Pharma in France. At Ariana (2019-2026), he led the alignment of AI product development and regulatory strategies of Ariana Pharma customers. He led MBQ Pharma (2018-2022) in the development and IND acceptance by the FDA of a first-in-class Rac/CDC42 metastasis removal therapy. Before Ariana, he was VP of Product Development and Regulatory at TOMA Biosciences (2015-2017), where he coordinated the development and CDRH FDA pre-submission acceptance of a 130-gene NGS oncology panel. He was VP for Strategic Regulatory Intelligence at Vertex Pharmaceuticals (2011-2015), where he transformed informal Voluntary Exploratory Data Submission (VXDS) meetings with CDER into a tool for an in vitro surrogate biomarker for Kalydeco efficacy and the subsequent expansion of the Kalydeco label for 24 additional CFTR mutations and 900 Cystic Fibrosis patients. At the US FDA (2004-2011), he led over 40 VXDS meetings with different pharmaceutical industry companies, developed the Biomarker Qualification Process for the universal acceptance of biomarkers by the FDA, and started, together with colleagues at NCTR, the MicroArray Quality Consortium (MAQC). His earlier seminal work at Applied Biosystems (1996-2000) led to the development of Precision Medicine platforms with the first TaqMan and SYBR qPCR master mixes and pre-developed assays, and their subsequent application in drug safety evaluation at Schering-Plough (2000-2004).