Niemann-Pick disease research is one of the pillars of our mission, and we are proud that since 1993, NNPDF has funded over 50 grants and 22 fellowships totaling over $4,000,000. Our fellowships and grants have contributed to the identification of disease-causing genes, insights into the pathogenesis of NPC1 & NPC2, support of the NPC cat model at a critical juncture, and development of an assay for newborn screening. Many former grantees and fellows have remained active contributors in the Niemann-Pick disease space.
NNPDF remains committed to advancing research that improves our understanding of Niemann-Pick disease and supports the development of potential treatments.
While the Foundation is not currently offering an open research grant or fellowship funding cycle, research proposals may be considered periodically on a case-by-case basis, depending on available funding and alignment with NNPDF’s research priorities.
Researchers interested in potential funding opportunities are encouraged to contact Executive Director Heather Teal for additional information regarding proposal consideration and application requirements.
Explore our historical record of research grants and fellowships, including projects supported in collaboration with research and funding partners.
These records have been compiled from NNPDF’s published research listings and archived foundation documents.
Listed by documented start or award date, most recent first.
| # | Recipient | Project title | Dates |
|---|---|---|---|
| 52 | Melissa Wasserstein, MD | Niemann-Pick disease: Genotype/Phenotype Analyses and Molecular Based Therapy | Oct 2014 – Oct 2018 |
| 51 | Charles Vite, DVM, PhD | NPC Cat Colony “Fellowships of the Cats II” | Aug 2013 – Jul 2015 |
| 50 | Charles Vite, DVM, PhD | NPC Cat Colony “Fellowships of the Cats I” | Aug 2011 – Sep 2013 |
| 49 | Jackson Laboratory Repository | Processing the NPD Mouse Model Developed by Dr. Richard Pagano of Mayo Clinic College of Medicine | Aug 2009 |
| 48 | Edward H. Schuchman, PhD | Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick | Apr 2008 – Mar 2009 |
| 47 | Frances M. Platt, PhD & Emyr Lloyd-Evans, PhD | Targeting Sphingosine Storage as a New Therapeutic Approach for NPC | Mar 2008 – Feb 2010 |
| 46 | Lysosomal Storage Disease Research Consortium | Additional Contribution to LSD Research Consortium | Apr 2007 – Mar 2008 |
| 45 | Frances M. Platt, PhD | Evaluation of Anti-Inflammatory Intervention (NSAIDs) as Adjunctive Therapy in NPC1 Disease | Mar 2007 – Feb 2008 |
| 43 | Maria D. Ledesma, PhD | Analysis of Dendritic Spine Alterations in NPD Type A Neurons | Sep 2006 – Aug 2007 |
| 42 | Kostantin Dobrenis, PhD | Establishment of CNS Cell Lines from NPC-1 Disease Mice for Pathogenic/Therapeutic Studies | Jun 2006 – Dec 2007 |
| 41 | Frances M. Platt, PhD | Therapeutic Monitoring of Glycolipid Storage Diseases Using Cell Biological Assays and Proteomics | May 2006 – Nov 2006 |
| 44 | Lysosomal Storage Disease Research Consortium | Additional Contribution to LSD Research Consortium | Apr 2006 – Mar 2007 |
| 38 | Thomas J. Kelley, PhD | NOS2 Regulation in NPC | May 2005 – Apr 2006 |
| 39 | Robert A. Maue, PhD | Maintenance of Colony of NPC1 Mice with GFP-Tagged Purkinje Neurons | Apr 15, 2005 – Apr 14, 2006 |
| 37 | Richard E. Pagano, PhD & David L. Marks, PhD | Reduction of Stored Lipids in NPC Mice by RAB Protein Expression | Apr 2005 – Apr 2007 |
| 36 | Maria D. Ledesma, PhD | Organization of Synapses in Niemann-Pick Disease Type A | Sep 2004 – Sep 2005 |
| 40 | Lysosomal Storage Disease Research Consortium | Contribution for NNPDF to Join the LSD Research Consortium | Apr 2004 – Mar 2006 |
| 35 | Synthia Mellon, PhD | SSRIs as a Potential Therapy for NPC | Apr 2004 – Mar 2005 |
| 34 | Stuart A. Lipton, MD, PhD | NMDA Receptor Hypofunction in Niemann-Pick Disease Type A | Mar 2004 – Feb 2005 |
| 31 | Maria D. Ledesma, PhD | NPD Type A: Molecular Analysis of Neuronal Membrane Maturation and Synapse Formation | Awarded Aug 15, 2002 |
| 33 | Makoto Michikawa, MD, PhD | Cellular and Molecular Mechanisms Underlying… | Est. 2002–2003 |
| 32 | Timothy M. Cox, MD & Robin H. Lachmann, PhD | Therapeutic Gene Delivery to the Cerebellum in Niemann-Pick Disease | Est. 2002–2003 |
| 30 | Anita H. Corbett, PhD | Functional Analysis of NPC1 | Awarded Jan 2002 |
| 29 | Melanie Jean Dobson, PhD | A Yeast System for Analysis of Sterol Trafficking Defects in NPC | Awarded Jan 2002 |
| 28 | Daniel S. Ory, MD | The Role of HE1 in Cholesterol Trafficking | Awarded Aug 2001 |
| 27 | Skadrite Krisans, PhD | Role of Peroxisomal Cholesterol/Myelin Formation in the CNS Pathology of the NPC Mouse | Awarded Aug 2001 |
| 26 | Anthony H. Futerman, PhD | Neuronal Development & Death in Type A Niemann-Pick Disease | Awarded Feb 2001 |
| 25 | Mary Anna Thrall, DVM | Characterization of Feline Niemann-Pick Type C | Awarded Aug 2000 |
| 24 | Yiannis A. Ioannou, PhD | Functional Characterization of NPC1L1, a Human Homologue of NPC1 | Awarded Aug 2000 |
| 23 | Robert A. Maue, PhD | Analysis of Growth Factor Actions and Membrane Polarity in Neurons from NP-C Mice | Awarded Aug 2000 |
| 22 | Daniel S. Ory, MD | Molecular Cloning of the Niemann-Pick Type C2 Gene | Awarded Apr 2000 |
| 20 | Lisa D. Griffin, MD, PhD | Altered Neurosteroidogenesis in NPC Disease | Awarded Apr 2000 |
| 21 | Cynthia J. Forehand, PhD | Glutamate Receptors and Neuronal Degeneration in NPC Disease | Awarded Feb 2000 |
| 19 | James Shayman, MD | Treatment of NPC Disease with Potent Inhibitors of Cerebroside Synthase | Awarded Feb 2000 |
| 18 | Leslie P. Henderson, PhD | Analysis of NPC1 mRNA Expression in the Mouse Brain | Awarded Aug 1999 |
| 17 | Daniel Ory, MD | Molecular Cloning of NPC2 Gene | Awarded Jul 1999 |
| 16 | Lisa D. Griffin, MD, PhD | The Role of Neurosteroids in the Development of NPC | Awarded Jun 1999 |
| 15 | Frederick R. Maxfield, PhD | Intracellular Cholesterol Trafficking in NPC Disease | Awarded Jun 1999 |
| 14 | Jerry Reagan, PhD | Acid Sphingomyelinase Regulates Cholesterol Egress from Lysosomes by an Alternative Pathway | Awarded Sep 1998 |
| 13 | Daniel Ory, MD | Molecular Cloning of NPC2 Gene | Awarded Apr 1998 |
| 11 | Ira Tabas, MD, PhD | Phospholipid Metabolism and Cholesterol-Induced Cytotoxicity in NPC Macrophages | Awarded Mar 1998 |
| 10 | Leslie P. Henderson, PhD | Analysis of NPC1 mRNA Expression in the Mouse Brain | Awarded Mar 1998 |
| 12 | Jill Morris, PhD | Identification of Additional NPC1 Mutations | Awarded Feb 1998 |
| 7 | Marc Patterson, MD | Establish an International Registry (database) of NPC Families | Awarded Mar 1997 |
| 9 | Marc Patterson, MD | Lipid Trafficking in the Neurons of NPC Mice | Awarded Jan 1997 |
| 8 | Synthia Mellon, PhD | Potential Involvement of Neurosteroids in Neurobiological Dysfunctions of NPC | Awarded Oct 1996 |
| 6 | G. S. Tint, PhD | Accumulation of Oxidized Cholesterol as a Possible Cause of NPC Pathologies | Awarded Jul 1996 |
| 5 | John R. Sargent, PhD | Influence of Exogenous Fatty Acids on Cholesterol in Cultured Skin Fibroblasts from NPC Patients | Awarded Jun 1996 |
| 4 | Wenda L. Greer, PhD | Identification of Niemann-Pick Type D Patients with Acadian Ancestry | Awarded Jan 1996 |
| 3 | Charles C. Schwartz, MD | Quantitation of Cholesterol Transport and Tissue Cholesterol Pool Sizes In Vivo NPC Disease | Awarded Nov 1995 |
| 2 | Laura Liscum, PhD | Complementation Analyses of NPC Fibroblasts’ Cholesterol Transport in Defective CHO Cells | Awarded Apr 1994 |
| 1 | Eugene Carstea, PhD | Identify the Gene Responsible for Niemann-Pick Type C Disease | Awarded Jul 1993 |
Newest to oldest, with original fellowship numbers preserved.
| # | Fellow | Project title | Dates |
|---|---|---|---|
| 22 | Bilal Abdul Moiz | Membrane Deficiency in NPC and Brain Endothelial-Mediated Glucose Supply to the Brain | Oct 2022 – Oct 2023 |
| 21 | Sara Naya Forcano | Intranasal Delivery of Chitosan-Encapsulated Hydrocortisone for ASMD Brain Pathology | Oct 2022 – Oct 2023 |
| 20 | Steven Erwood | Generation of Humanized Mouse Models for NPC Disease | Sep 2020 – Aug 2021 |
| 19 | Christian Kappe (PhD student) | In Cellulo Characterization of Small Molecule Activators of Acid Sphingomyelinase | Sep 2020 – Mar 2022 |
| 18 | Adam Kanack | Prevention of NPC1 Ubiquitination as a Therapeutic Target for NPC Disease | Oct 2016 – Oct 2019 |
| 17 | Ofer Moldavski, PhD | NPC Disease as an mTORC1-Dependent Metabolic Disease | Jan 2016 – Dec 2018 |
| 16 | Xuntian Jiang, PhD | Development of a Diagnostic Test and Newborn Screen for NPC Disease | Jan 2016 – Jan 2017 |
| 15 | Andrew Munkacsi, PhD | Molecular Basis of HDAC Inhibitors (Yr 3 extension) | May 2015 – Nov 2015 |
| 14 | Gizely Andrade | Neurophysiological Outcome Measures for Treatment Assessment in NPC Disease | Oct 2014 – Oct 2016 |
| 13 | Xuntian Jiang, PhD | Development of a Diagnostic Test and Newborn Screen for NPC Disease | Oct 2014 – Oct 2015 |
| 12 | Kanagaraj Subramanian | Roadmap for Drug Development for Niemann-Pick disease | Oct 2014 – Oct 2016 |
| 11 | Deepti Gadi, PhD | Cellular Mechanism of HDAC Inhibitors for Treatment of NPC Disease | Oct 2012 – Oct 2014 |
| 10 | Stephanie M. Cologna, PhD | Differential Protein Expression Profiling in CSF from NPC1 Patients | Oct 2012 – Oct 2014 |
| 9 | Lluis Samaranch Gusi, PhD | AAV9-Mediated Human Acid Sphingomyelinase Expression in Nonhuman Primate Brain (Gene Therapy for NP Type A) | Oct 2012 – Oct 2014 |
| 8 | Andrew Munkacsi, PhD | Molecular Basis of HDAC Inhibitors to Treat Murine, Feline & Human Models of NPC (Yrs 1–2) | Sep 2011 – Feb 2014 |
| 7 | Dorothea Maetzel, PhD | In Vitro Modelling of NPC Disease Using Patient-Specific iPSCs | Sep 2010 – Sep 2013 |
| 6 | Nicholas Cianciola, PhD | Activation of an Alternative Cholesterol Homeostatic Mechanism in NPC | Sep 2010 – Sep 2013 |
| 5 | Fabrizio Vacca, PhD | Analysis of Cholesterol Export from Purified Endosomes in NPC Cellular Models | Apr 2010 – Apr 2012 |
| 4 | Ian Williams, PhD | Neurobiology of Purkinje Cells in NPC1 | Sep 2009 – Sep 2011 |
| 3 | Nobutaka Ohgami, PhD | Demonstration of NPC1 as a Lipid Binding Protein in Vitro | Sep 2004 – Dec 2007 (extended) |
| 2 | Heng-Ling Liou, PhD | The Role of NPC2 Protein in Lysosomal Cholesterol Trafficking | Mar 2003 – Mar 2005 |
| 1 | Amit Kumar Choudhury, PhD | Reduction of Lipid Accumulation in Niemann-Pick Fibroblasts by Modulation of Membrane Trafficking | Mar 2003 – Mar 2005 |
The Researcher Travel Program is intended to assist with travel expenses for rare disease researchers and professional students with an interest in the rare disease field. It is our hope that this program provides opportunities for these individuals to connect in person with the rare disease community and to foster collaboration and communication.
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