As of July 1, 2019

Pei Zhao, Research Associate, The Scripps Research Institute -Title: Management of NPC1 Variation in the Clinic

Brian Blagg, Professor, Director of the Warren Family Research Center for Drug Discovery and Development, University of Notre Dame – Title: Investigations of NPC Disease and Lysosomal Storage Disorders through Computational and Medicinal Chemistry Studies

Eamonn Dickson, Assistant Professor, UC Davis – Title: Targeting Ion Channel Dysfunction as a Therapeutic for Neurodegeneration in NPC1

Daniel Heller, Assistant Member, Memorial Sloan Kettering Cancer Center – Title: In Vivo Reporter for NPC disease Drug Discovery

Paul Helquist, Professor, University of Notre Dame – Title: Actively Targeted BBB-Crossing Cyclodextrin Derivatives for Treatment of NPC

Yiannis Ioannou, Professor, Icahn School of Medicine at Mount Sinai – Title: Reduction of ER stress as a strategy for the treatment of NPC1 disease: proof of concept by targeting PDIA3

Fred Maxfield, Professor, Cornell – Title: Systematic examination of the role of chaperones in folding the NPCl protein

Paulina Ordonez, Assistant Professor, University of California, San Diego – Title: Metabolic characterization and pharmacodynamics of mitoprotective compounds to advance and FDA approved treatment for NPC

Bill Pavan, National Institutes of Health – Title: GPNMB as a biomarker for NPC1 disease progression and therapeutic assessment; Two-year

Suzanne Pfeffer, Professor of Biochemistry, Stanford University – Title: NPC1-mediated export of cholesterol from lysosomes

Forbes D. Porter, Senior Investigator and Clinical Director, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health – Title: Cerebellar transcriptome analysis by single cell RNA sequencing

Kevin Vaughan, Associate Professor, Notre Dame – Stimulation of StARD9 as a Novel Mechanism to Overcome NPC Disease.

Charles Vite, Professor, University of Pennsylvania – Improving Extracerebellar NPC1 Disease using Intravenous 2-Hydroxypropylbeta-Cyclodetrin

Nieng Yan, Shirley M. Tilghman Professor of Molecular Biology, Princeton university – Structure and mechanistic elucidation of NPC1 and NPC2-mediated cholesterol egress

Hongyuan Yang, Professor, University New South Wales – Title:  The role of ORP1L, ORP2 and phosphoinositides in NPC1 function