Special seminar by Prof. Frances Platt

10.8.2026

Päivämäärä
10.08.2026
Aika
13:00 - 14:00
Sijainti
Biomedicum Helsinki 1, lecture hall 3
Organisaatio
University of Helsinki, Faculty of Medicine
Yhteyshenkilö
thomas.mcwilliams@helsinki.fi

Prof. Frances Platt FMedSci FRS, University of Oxford

Niemann-Pick Disease Type C: From Molecular Pathways to Therapeutic Impact

About Fran

Fran is a biochemist and pharmacologist internationally recognised for her pioneering contributions to lysosomal storage disorders and the role of lysosomes in progressive neurodegenerative disease and beyond. She has established several important concepts in the field, with major expertise on glycosphingolipids (GSLs) and lysosomal storage disorders. Fran’s research has led to the development of approved substrate reduction therapies for Niemann-Pick disease type C and Gaucher disease, with additional therapeutic strategies currently in clinical trials. Her research combines neuroscience, lysosomal cell biology and lipid biochemistry, spanning preclinical animal and human models, as well as therapeutic studies. Fran originally completed her BSc in Zoology at Imperial College London and her PhD in Animal Physiology at the University of Bath, before undertaking postdoctoral research at Washington University School of Medicine, St Louis, MO, USA. Following a period in industry, Fran moved to the University of Oxford, where she is Professor of Biochemistry and Pharmacology and currently Head of the Department of Pharmacology, as well as a supernumerary Fellow at Merton College. In recognition of her outstanding contributions to science, she has received numerous awards and was elected a Fellow of the Academy of Medical Sciences in 2011, a Member of Academia Europaea in 2018 and a Fellow of the Royal Society in 2021.

Fran is also due to be a keynote speaker at the 2026 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), which will also take place in Helsinki from August 25–28. We are therefore especially fortunate to have this opportunity to hear from her at the Faculty.

I warmly encourage colleagues (especially students and postdocs) across the Faculty and wider biomedical community to attend, as this is a rare opportunity to hear from a scientist of genuine distinction.

A limited number of meeting slots are available. For a meeting with Professor Platt, please contact me directly.

Looking forward to welcoming you to the seminar!

With warm regards

Tom McWilliams

Selected recent papers

Höglinger D, Burgoyne T, Sanchez-Heras E, Hartwig P, Colaco A, Newton J, Futter CE, Spiegel S, Platt FM, Eden ER. NPC1 regulates ER contacts with endocytic organelles to mediate cholesterol egress. Nat Commun. 2019 Sep 19;10(1):4276. doi: 10.1038/s41467-019-12152-2. PMID: 31537798; PMCID: PMC6753064.

Platt FM. The expanding boundaries of sphingolipid lysosomal storage diseases; insights from Niemann-Pick disease type C. Biochem Soc Trans. 2023 Oct 31;51(5):1777-1787. doi: 10.1042/BST20220711. PMID: 37844193; PMCID: PMC10657176.

Weng Y, Shepherd D, Liu Y, Krishnan N, Robertson BD, Platt N, Larrouy-Maumus G, Platt FM. Inhibition of the Niemann-Pick C1 protein is a conserved feature of multiple strains of pathogenic mycobacteria. Nat Commun. 2022 Sep 9;13(1):5320. doi: 10.1038/s41467-022-32553-0. PMID: 36085278; PMCID: PMC9463166.

Platt FM, d’Azzo A, Davidson BL, Neufeld EF, Tifft CJ. Lysosomal storage diseases. Nat Rev Dis Primers. 2018 Oct 1;4(1):27. doi: 10.1038/s41572-018-0025-4. PMID: 30275469.