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Niemann-Pick disease
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#	Item
21	NNPDF-Funded Research Grant # 47 TITLE: Targeting sphingosine storage as a new therapeutic approach for Niemann-Pick type C PROJECT INVESTIGATOR: Fran Platt & Emyr Lloyd-Evans PERIOD: [removed]2010 PROJECT DESCRIP Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:40:00 Lipid storage disorders Lipids Rare diseases NPC1 Membrane biology Sphingomyelin Niemann–Pick disease Sphingolipid Miglustat Chemistry Biology Health
22	September 30, 2013 Dr. Charles Vite University of Pennsylvania Veterinary School of Medicine NNPDF Research Grant ~ Final Report August 2011 thru September 2013 Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2013-10-07 17:48:43 Routes of administration Biomarkers Chemical pathology NPC1 Intrathecal Miglustat Niemann-Pick disease type C Medicine Pharmacology Dosage forms
23	NNPDF-Funded Research Grant # 48 TITLE: Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick PROJECT INVESTIGATOR: Edward H. Schuchman, PhD PERIOD: [removed]2009 PROJECT DESCRIPTION Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:40:23 Health Niemann–Pick disease Lysosomal storage disease Sphingomyelin phosphodiesterase Sphingomyelin Lipid storage disorders Biology Rare diseases
24	A Brief History of NNPDF-Supported Research NNPDF was the first non-profit, family-based organisation to fund research on Niemann-Pick Disease As we celebrate the 15th Anniversary of NNPDF, the Foundation is recognising Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2007-08-02 16:12:53 NPC1 Epididymal secretory protein E1 Niemann–Pick disease Niemann-Pick disease type C Lipid storage disorders Rare diseases Health
25	NNPDF COLLABORATIVE STUDY – KAREN QUANDT, RN, MN Neurodegenerative Disease in Family Members of Patients with Niemann-Pick Type C Final Progress Report – Lay Summary Thank you to all the families that participated in Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2011-06-22 13:11:55 Niemann–Pick disease Motor neurone disease Degenerative disease Amyotrophic lateral sclerosis Niemann-Pick disease type C Epididymal secretory protein E1 Health Rare diseases Lipid storage disorders
26	National Niemann-Pick Disease Foundation, Inc. 401 Madison Avenue, Suite B; Post Office Box 49 Fort Atkinson, WI[removed][removed]; Fax #: [removed]Web Site: www.nnpdf.org; E-mail: [removed] Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2012-01-18 10:41:09 Fax Internet Computing Technology Email Press release
27	Orphanet Journal of Rare Diseases This Provisional PDF corresponds to the article as it appeared upon acceptance. Fully formatted PDF and full text (HTML) versions will be made available soon. Niemann-Pick disease type Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-06-08 17:44:24 Rare diseases NPC1 Niemann–Pick disease Niemann-Pick disease type C Lysosomal storage disease Epididymal secretory protein E1 Hepatosplenomegaly Cataplexy Splenomegaly Health Medicine Lipid storage disorders
28	National Niemann-Pick Disease Foundation Grant #48 Six-Month Progress: Lay Summary – Edward H. Schuchman, PhD The goal of our research is to create a new mouse model of Type A NPD. This mouse will make an abnormal (or Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:40:14 Membrane biology Phospholipids Sphingomyelin Niemann–Pick disease Lysosome Lysosomal storage disease Niemann-Pick disease SMPD1-associated Sphingomyelin phosphodiesterase 1 Biology Lipid storage disorders Rare diseases
29	NNPDF-Funded Research Grant # 43 TITLE: Analysis of Dendritic Spine Alterations in Niemann-Pick Disease Type A Neurons PROJECT INVESTIGATOR: Maria D. Ledesma, Ph.D. PERIOD: [removed]2007 PROJECT DESCRIPTION Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-04-26 15:39:24 Neurophysiology Neurons Cellular neuroscience Lipid storage disorders Dendritic spine Sphingomyelin Niemann–Pick disease Dendrite Lysosome Biology Neuroscience Nervous system
30	\|Barb Vorpahl FDA Presentation January 12, 2010 Good Afternoon. My name is Barb Vorpahl, and I am chair of the National Niemann-Pick Disease Foundation. My husband and Gary and I also had a daughter, Stacey who died at t Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-01-14 15:59:00 Medicine Neurodegenerative disorders Niemann–Pick disease Miglustat Cataplexy Health Lipid storage disorders Rare diseases