• Christopher Austin, M.D. – Director, National Institutes of Health, Chemical Genomics Center – TITLE:  “Identifying a Potential New Therapy for NP-C Patients by Screening of FDA-Approved Drugs”
  • David Azorsa, Ph.D. – Senior Investigator, Pharmaceutical Genomics, Translational Genomics Research Institute (TGen) – TITLE:  “Application of Functional Genomics to Identify and Validate Genes that Restore Normal Cholesterol Transport in NP-C Cells”
  • Philip Beachy, Ph.D. – Associate Professor, Department of Molecular Biology and Genetics, Johns Hopkins University School of Medicine – $220,000 award (2-year grant from 1/98 – 12/99) TITLE: “Cholesterol homeostasis and NP-C: A role in cell signaling?”
  • Krishna Bhat, Ph.D. – Emory University School of Medicine – $208,647 award (2-year grant from 7/01 – 6/03) TITLE:  “NPC disease in Drosophila mutant for the tumor suppressor Patched”
  • Kendal Broadie, Ph.D. – Assistant Professor, Department of Biology, University of Utah – $250,000 award (2-year grant from 1/00 – 12/01) TITLE: “Neurological functions of NPC1 in Drosophila”
  • Maja Bucan, Ph.D. – Assistant Professor, College of Medicine, University of Pennsylvania – $250,000 award (2-year grant from 7/95 – 6/97) TITLE: “Genetic Mapping and Isolation of the mouse NP-C Disease Gene”
  • Robert Burgess, Ph.D. and Kevin Seburn, Ph.D. – The Jackson Laboratory – $5,000 award (1-year grant from 4/06 – 3/07) TITLE:  “Characterization and development of a new mouse model of NP-C disease”
  • Eugene Carstea, Ph.D. – Senior Staff Fellowship Scientist, Developmental and Metabolic Neurology Branch, Neurology Institute, NIH – $250,000 award (2-year grant from 7/95 – 6/97) TITLE: “Integrative Efforts to Identify Gene for Niemann-Pick disease Type C”
  • Geoffrey Chang, Ph.D. – Associate Professor, The Scripps Research Institute – $96,000 award (1.5- year grant from 07/05-12/06) TITLE: “X-ray Structure of Niemann-Pick C1”
  • T.Y. Chang, Ph.D. – Professor of Biochemistry, Dartmouth Medical School – $151,844 award (2-year grant from 1/00 – 12/01) TITLE: “Role of NPC1 in Intracellular Cholesterol Trafficking”
  • David Craig, Ph.D. – Associate Investigator, Neurogenomics, Translational Genomics Research Institute (TGen) – $47,073 award(1-year grant from 7/06 – 6/07)  – TITLE:  “Identification of the mechanism of allopregnanolone-mediated neuroprotection in Niemann-Pick Type C disease”
  • Kumlesh Dev, Ph.D. –Laboratory Head of Molecular and Cellular Neuroscience, Novartis Pharma A.G., Switzerland – TITLE: “Identification of Molecular Targets Linking Alzheimer’s disease and Niemann-Pick Type C disease.”
  • Robert Erickson, M.D. – Professor of Human Genetics and Inherited Diseases, University of Arizona College of Medicine – $141,666 award (2.5-year grant from 7/97 – 12/99) TITLE: “Cloning the Mouse Homolog of the Niemann-Pick C Gene”
  • William Garver, Ph.D. – Research Assistant Professor, Department of Pediatrics, University of Arizona – $285,009 award (5.5-year grant from 1/01 – 6/06) TITLE: “Research Projects for the Investigation of Niemann-Pick Type C disease”
  • Robert Gillies, Ph.D. – Professor, Biochemistry, University of Arizona College of Medicine – $157,465 award (2-year grant from 1/99 – 12/00) TITLE: “Non-invasive Monitoring of NP-C Progression and Therapy with MRS/MRI”
  • Wenda Greer, Ph.D. – Associate Professor, Pathology Department, Dalhousie University, Halifax, Nova Scotia – $75,000 award (1-year grant from 8/96 – 7/97)
    TITLE: “Localizing the Niemann-Pick D Gene (Nova Scotia Variant of Type C)”
  • Richard Hawkes, Ph.D. – Professor, Department of Cell Biology & Anatomy, University of Calgary – $152,760 award (2-year grant from 1/00 – 12/01) TITLE: “Patterned Purkinje Cell Loss in the Cerebellum of a Murine Model of NPC: A Possible Neuroprotective Role for the Small Heat Shock Protein HSP25”
  • Elina Ikonen, M.D., Ph.D. – Senior Scientist of the Academy of Finland, National Public Health Institute, Helsinki, Finland – $347,500 award (2-year grant from 1/99 – 12/00) (1.5-year grant from 7/02 –12/03) (additional grant from 1/00 – 12/01) TITLE: “NPC lesion at the crossing of cellular cholesterol trafficking circuits”
  • Rodney Infante, Ph.D. – NP-C Fellowship – UT Southwestern Medical Center at Dallas – TITLE:  “Biochemical Characterization of NPC1 and NPC2”
  • Yiannis Ioannou, Ph.D. – Mt. Sinai School of Medicine – $250,000 award (2-year grant from 7/02 – 6/04) (Additional 6 years of awards totaling $719,902 from 3/96 – 6/02) TITLE:  “Cellular and Molecular Studies of Niemann-Pick C Disease”
  • Barbara Karten, Ph.D. – Assistant Professor, Biochemistry and Molecular Biology, Dalhousie University, Canada – TITLE:  “The Role of NPC1 in Synaptic and Neuronal Function”
  • Toshihide Kobayashi, Ph.D. – Group Leader, Sphingolipid Functions Laboratory, RIKEN Frontier Research System, Japan – $125,000 award (1-year grant from 1/01 – 12/01) TITLE: “Effects of cholesterol on endocytic pathway in NPC cells”
  • Jeffrey Krise, Ph.D. – Assistant Professor, The University of Kansas – $125,000 award (1-year grant from 7/06 – 6/07) TITLE:  “The Role of NPC1 in the intracellular trafficking of endogenous amines”
  • Yvonne Lange, Ph.D. – Professor, Department of Pathology, Rush Presbyterian – St. Lukes Medical Center – $103,796 award (1-year grant from 1/98 – 12/98) TITLE: “A Novel Treatment of Niemann-Pick Type C disease”
  • Laura Liscum, Ph.D. – Professor, Physiology, Tufts University School of Medicine – $295,690 award (3.5-year grant from 8/97 – 12/00) TITLE: “Analysis of pharmaceutical compounds that correct the NPC phenotype in cultured cells”
  • Emyr Lloyd-Evans, Ph.D. – Department of Biology, Oxford University – $46,000 award (6-month award during Fall 2005) TITLE:  “Lysosomal Calcium Homeostasis Defect in NPC1 Cells that contribute to Endocytic Defects in NPC1”
  • Benny Liu, M.D. – NP-C Fellowship, UT Southwestern Medical Center at Dallas – TITLE: “Molecular Events Leading to Cell Death in the NP-C Syndrome”
  • Robert Maue, Ph.D. – Associate Professor, Departments of Physiology and Biochemistry, Dartmouth Medical School – $279,950 award (2.5-year grant from 7/97 – 12/99) TITLE: “Striatal and hypothalamic neuron function in NP-C mice”
  • Synthia Mellon, M.D. – Professor, Department of Obstetrics, Gynecology and Reproductive Sciences, University of California, San Francisco – $537,500 award (4.5-year grant from 1/01 – 6/05) TITLE:  “Therapeutic Strategies for Treatment of NP-C”
  • James Metherall, Ph.D. – Assistant Professor, Department of Human Genetics, University of Utah – $275,894 award (3-year grant from 8/95 – 7/98) TITLE: “Genetic Defects in Proteins Required for Intracellular Cholesterol Transport Cause NP-C”
  • Gilles Millat, Ph.D. – Assistant Professor, INSERM and Lyon University, France – $112,200 award (2-year grant from 1/03 – 12/04)  TITLE:  “Generation of mice with specific mutations affecting the cysteine-rich luminal loop of the NPC1 protein”
  • Haruaki Ninomiya, M.D. – Associate Professor, Neurobiology, Tottori University Faculty of Medicine, Japan – $50,000 award (1-year grant from 7/06 – 6/07)  TITLE:  “Quality Control and degradation mechanisms of NPC1”
  • Axel Nohturfft, Ph.D. – Assistant Professor, Department of Molecular and Cellular Biology, Harvard University – $394,463 award (4.5-year grant from 1/01 – 6/05) TITLE:  “Role of apolipoprotein E in Niemann-Pick disease Type C”
  • Daniel S. Ory, M.D. – Associate Professor, Internal Medicine, Washington University – TITLE: “Chemical Chaperone Therapy for NP-C Disease”
  • Richard Pagano, Ph.D. and David Marks, Ph.D. – Professors, Department of Biochemistry & Molecular Biology, Mayo Clinic Rochester, MN – $708,349 awarded (7-year grant period from 1/98 – 6/04) TITLE: “Correction of NP-C Phenotype by Rab Overexpression”
  • Leo Pallanck, Ph.D. – Assistant Professor, Genome Sciences, University of Washington – $306,866 award (4-year grant period from 7/02 – 6/06) TITLE:  “Development of a Drosophila Model of Niemann-Pick Type C Disease”
  • William Pavan, Ph.D. – Section Chief, Laboratory of Genetic Disease Research, NIH – $246,060 award (3-year grant from 7/95 – 6/98) TITLE: “Exploiting Mouse Models to Identify the Gene for NP-C”
  • David Pearce, Ph.D. – Assistant Professor, Biochemistry and Biophysics, University of Rochester School of Medicine & Dentistry – $83,187 award – (1-year grant from 1/00 – 12/00) TITLE: “A Yeast Model for Studying Niemann-Pick Type C”
  • Suzanne Pfeffer, Ph.D. – Professor, Biochemistry, Stanford University School of Medicine – TITLE: “Molecular Analysis of  NPC1 Protein”
  • Frank Pfrieger, Ph.D. – Assistant Professor, Centre de Neurochimie, Stasbourg, France – $200,000 award (2-year grant from 7/02 – 6/04) TITLE: “Role of NPC1 in cholesterol shuttle from Glia to Neurons”
  • Frances Platt, Ph.D. – Reader of Glycobiology, University of Oxford – $499,052 award (4-year award from 7/02 – 6/06) TITLE: “Cell Biology of Niemann-Pick C disease: Role of glycosphingolipids in Pathology”
  • Joyce Repa, Ph.D. – Assistant Professor, Physiology, UT Southwestern Medical Center at Dallas – TITLE:  “Nuclear Orphan Receptors as Targets for the Treatment of NP-C Disease”
  • Matthew Scott, Ph.D. – Professor, Developmental Biology, Stanford University School of Med. – TITLE: “Functions of the NPC1 Protein in Purkinje Neurons”
  • Frances Sharom, Ph.D. – Professor and Canada Research Chair, Molecular and Cellular Biology, University of Guelph, Canada – $407,858 award (5.5-year grant from 1/03 – 6/08) TITLE:  “Characterization of substrate binding, transport, lipid flippage and cholesterol transfer by the NPC1 protein”
  • James Shayman, M.D. – Professor of Internal Medicine, University of Michigan $102,947 award – (1-year grant from 3/00 – 2/01) TITLE: “Treatment of NPC-1 with Glucosylceramide Synthase Inhibitors”
  • Kyra L. Somers, D.V.M., Ph.D. – Assistant Professor, Microbiology, Immunology and Pathology, Colorado State University – $52,581 award (1-year grant from 7/2004 – 2/2005) TITLE:  “Feline Niemann-Pick C1 Disease”
  • Ann Stock, Ph.D. – Professor, UMDNJ-Robert Wood Johnson Medical School Center for Advanced Biotechnology & Medicine & Associate Investigator, HHMI – $530,556 award (4.5-year grant from 1/03 – 6/07) TITLE: “Structural Analysis of NPC2”
  • Judith Storch, Ph.D. – Professor, Nutritional Sciences, Rutgers University – TITLE: “Mechanisms of NPC2 Protein in Cholesterol Transport”
  • Jerome Strauss III, Ph.D. – Professor, Obstetrics and Gynecology, University of Pennsylvania – $313,654 award (6-year award from 1/98 – 12/03) TITLE: “Function of MLN 64”
  • Stephen Sturley, Ph.D. – Assistant Professor, Institute of Human Nutrition, Columbia University – $1,320,000 award (10-year grant from 8/97 – 6/07) TITLE: “A genetic model of Niemann-Pick Type C disease”
  • Steve Sturley, Ph.D. – Associate Professor, Columbia University – TITLE:  “Modulating Niemann-Pick Type C Disease in Yeast, Mice and Humans”
  • Derek Symula, Ph.D. – Research Scientist IV, Genomics Institute Wadsworth Center – TITLE: “Genetic Modifiers of NPC2 Deficiency in Mice.”
  • Kinuko Suzuki, M.D. – Professor of Pathology and Laboratory Medicine, University of North Carolina – $203,247 award (2-year grant from 1/00 – 12/01)
    TITLE: “Neuronal death and glial cell reactions in NP-C mouse”
  • Danilo Tagle, Ph.D. – Head – Molecular Neurogenetics Section, NIH – $83,000 award (1-year grant from 8/97 – 7/98) TITLE: “Characterization of the Niemann-Pick Type C Gene Product (NPC1)”
  • Alan Tall, M.D. – Professor in Medicine, Columbia University – $200,200 award (2-year grant from 1/98 – 12/99) TITLE: “Atherosclerosis and Reverse Lipid Transport in NPC”
  • James Thomas, Ph.D. – Professor, Department of Genetics, University of Washington – $120,000 award (2-year grant from 7/1/00 – 6/30/02) TITLE: “Genetic and Cellular Analysis of a C.elegans Model for Niemann-Pick Type C disease.”
  • David H. Thompson, Ph.D. – Professor of Chemistry, Purdue University – TITLE: “Impact of HPbCD Polyrotaxanes on Inflammation in the NPC Mouse”:
  • Ted Trouard, Ph.D. – Associate Professor, Biomedical Engineering and Radiology, University of Arizona, – $17,000 award (Spring 2006) TITLE:  “Non-invasive Imaging to Detect and Monitor the Progression of NPC Disease and its Response to Therapy”
  • Gerrit van Meer, Ph.D. – Professor, Membrane Enzymology, Utrecht University, The Netherlands – TITLE: “NPC1 Pumping Simple Glycosphingolipids”
  • Jean Eaton Vance, Ph.D. – Professor, Heritage Medical Research Center, University of Alberta, Canada – $782,936 award (5.5 year grant from 1/00 – 6/06) TITLE: “Role of NPC1 in Primary Cultures of Murine Neurons”
  • Kevin Vaughan, Ph.D. and Holly Goodson, Ph.D. – Associate and Assistant Professors, Biological Sciences, University of Notre Dame – $150,000 award (2-year grant from 7/06 – 6/08) TITLE: “Microtubule-Dependent Transport of NPC-Containing Membranes”
  • Inez Vincent, Ph.D. – Research Associate Professor, Department of Pathology, University of Washington – $45,000 award (1-year grant from 1/1/03 – 12/31/03) TITLE:  “Cdk inhibitors for treating NPC.”
  • Steven Walkley, D.V.M., Ph.D. – Professor, Neuroscience, Albert Einstein College of Medicine – TITLE:  “Gangliosides in the Pathogenesis of  NP-C”
  • NP-C Genetic Counseling and Carrier Testing Center – Mayo Clinic, Rochester, MN – genetic counselor, Cate Walsh-Vockley
  • Silvana Zanlungo, Ph.D. – Assistant Professor, Gastroenterology, Pontificia Universidad Catolica de Chile – TITLE:  “The Proapoptotic c-Abl Kinase as a Therapeutic Target for NP-C Disease”
  • Hongyuan Robert Yang, Ph.D., Professor and NHMRC Senior Research Fellow, School of Biotechnology and Biomolecular Sciences, University of New South Wales – TITLE: “A yeast model for studying NP-C1 and intracellular sterol transport”
  • Fabien Alpy, Ph.D., Researcher, French National Institute of Health and Medical Research (INSERM) – TITLE: The cholesterol transfer protein STARD3: a new therapeutic target in Niemann Pick C disease.
  • Gregorio Gil, Ph.D., Professor, Department of Biochemistry and Molecular Biology, Medical College of Virginia, Virginia Commonwealth University – TITLE: Activation of StarD4 corrects the NPC Phenotype.
  • Paul Helquist, Ph.D., Professor and Associate Chair for Research, Department of Chemistry and Biochemistry, The University of Notre Dame – TITLE: Design and Synthesis of Small Molecule Therapeutic Agents and Biochemical Probes for the Treatment and Study of Niemann-Pick Type C Disease.
  • Frederick R. Maxfield, Ph.D., Professor and Chair, Department of Biochemistry, Weill Cornell Medical College – TITLE: In Vitro tests of the efficacy of therapies for Niemann-Pick C disease.
  • Suzanne R. Pfeffer, Ph.D., Professor and Chairman of Biochemistry, Stanford University, School of Medicine – TITLE: NPC1-medicated export of cholesterol from lysosomes.
  • Kevin T. Vaughan, Ph.D., Associate Professor, Department of Biological Sciences, University of Notre Dame – TITLE: StARD9 as a Genetic Modifier of Niemann Pick Type C Disease.
  • Jing Zheng, Ph.D., Associate Professor, Department of Communication Sciences and Disorders, Northwestern University – TITLE: Investigate the Effectiveness of Salicylate in Preventing HPβCD-Induced Ototoxicity in the NPC1 Mouse Model.
  • Pei Zhao, Research Associate, The Scripps Research Institute -Title: Taming variation in NPC through proteostasis
  • Stephanie Cologna, Assistant Professor, University of Illinois-Chicago – Title: A mass spec approach to reveal proteomic and lipidomic patterns in progressive cerebellar neurodegeneration
  • Olga Ilnytska, Research Associate, Rutgers University – Title: Therapeutic Potential of LBPA enrighment in the treatment of NPC
  • Paulina Ordonez, Assistant Professor, University of California, San Diego – Title: Preclinical validation of mitoprotective compounds to advance an FDA-approved treatment for NPC
  • Suzanne Pfeffer, Professor of Biochemistry, Stanford University – Title: NPC-mediated export of cholesterol from lysosomes
  • Rich Taylor, Professor, University of Notre Dame – Title: Computational and medicinal chemistry studies of understanding NPC and LSDs development of new therapeutics
  • Andrew Lieberman, Gerald Abrams collegiate Professor, University of Michigan – Title: Optimization of sHDL nanoparticles for treatment of NPC
  • 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
  • Susanne Schneider, Professor, Ludwig-Maximilians-Universitat Munchen – Heterozygous mutations of NPC predispose to late-onset neurodegeneration – a clinical, radiological, and molecular character
  • 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