As of July 1, 2018

Pei Zhao, Research Associate, The Scripps Research Institute -Title: High Definition management of NPC1 variation through proteostasis-based redox system

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

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

Xuntain Jiang, Assistant Professor of Medicine, Washington University – Title: Oligosaccharides as a CNS biomarker for NPC disease

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

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: Development of hypomorphic NPC1 zebrafish and i3neurons to facilitate drug screening

Susanne Schneider, Professor, Ludwig-Maximilians-Universitat Munchen – Heterozygous mutations of NPC predispose to late-onset neurodegeneration – a clinical, radiological, and molecular character

Kevin Vaughan, Associate Professor, Notre Dame – Stimulation of StARD9 as a Compensatory Mechanism for NPC disease

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

Noam Zelcer, Professor of Molecular Regulation of Metabolism, Academic Medical Center of the University of Amsterdam – Interrogating cholesterol trafficking and NPC1 with mammalian haploid genetics

Roberto Zoncu, Assistant Professor of Biochemistry, Biophysics, and Structure Biology, UC Berkeley – Mechanisms for Lysosomal Cholesterol Sensing and Homeostasis

Mark Walterfang, Neuropsychiatrist and Professor of Neurosychiatry, Royal Melbourne Hospital – Resilience to NPC disease: Utilizing NPC superheroes to develop new therapeutic targets that buffer loss of function of NPC1

Pam Andrews, Firefly Fund – The Firefly Fund’s Newborn Screen Initiative for NPC1

Dan Ory, Alan A. and Edith L. Wolff Distinguished Professor of Medicine, Washington University – Title: Proteostatic regulation of treatment for NPC1 disease- Title: Optimization of sHDL nanoparticles for treatment of NPC

Charles Venditti and Bill Pavan, Senior Investigators, National Institutes of Health – Title:  AAV gene therapy for NPC