Opening Lecture - Sphingolipid Metabolism


Sphingolipid lysosomal storage diseases

Frances M. Platt is Professor of Biochemistry and Pharmacology and Head of the Department of Pharmacology, University of Oxford, UK.

She received her Ph.D. in Animal Physiology from the University of Bath, UK. After completing postdoctoral training at Washington University Medical School, St. Louis, she joined the faculty at the University of Oxford and was the recipient of a five-year Lister Institute Senior Research Fellowship. Her expertise relates to glycosphingolipids (GSL) and in particular glycosphingolipid (GSL) lysosomal storage diseases. She and her colleagues pioneered a novel approach to treat these inherited diseases that has led to the development of an approved drug (miglustat) for type 1 Gaucher disease and Niemann-Pick disease type C1 disease. She was awarded the Alan Gordon Memorial award from the UK Gaucher Association, the Horst-Bickel Award in recognition of her role in developing substrate reduction therapy for lysosomal disorders and the “Above and Beyond” award from National Tay-Sachs and Allied Diseases USA.

She has published extensively in this field over the past 20 years and co-edited a book titled Lysosomal Disorders of the Brain. She was an Editor for the Journal of Biological Chemistry (2009-2014) and serves on the advisory board of multiple lysosomal storage disease charities and organizations (UK and USA).

She was elected Fellow of the Academy of Medical Sciences in 2011 and Fellow of the Royal Society in 2021.

Frances M. Platt
University of Oxford, United Kingdom