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Niemann–Pick disease
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131	Microsoft Word - WORLD program FINAL[removed]doc Add to Reading List Source URL: www.lysosomaldiseasenetwork.org Language: English - Date: 2007-11-26 17:04:26 Cancer research Clinical research Medical genetics New York University School of Medicine Lysosomal storage disease Niemann–Pick disease Neurologist Robert J. Desnick Medicine Health Rare diseases
132	19 Black balls Rarely do we talk about them, rarely are there therapies for them, rarely do they even live to adulthood. Hope is often the only thing that remains for victims of rare diseases. 1.5 million zlotys. This i Add to Reading List Source URL: ec.europa.eu Language: English - Date: 2014-01-30 08:03:54 Lipid storage disorders Epidemiology Medical terms Neurodegenerative disorders Niemann–Pick disease Mucopolysaccharidosis Growth hormone treatment Disease Chronic Health Medicine Rare diseases
133	National Niemann-Pick Disease Foundation, Inc. P.O. Box 49; Fort Atkinson, WI[removed]USA) Tele: [removed]; Fax: [removed]Web Site: _www.nnpdf.org_ Email: [removed]_ Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2010-03-15 14:19:58 Email Niemann–Pick disease Niemann Lipid storage disorders Rare diseases Health
134	NEWS Niemann-Pick SPRING 2013 \| VOLUME 1, ISSUE 48 A publication of The National Niemann-Pick Disease Foundation (NNPDF) Add to Reading List Source URL: www.nnpdf.org Language: English - Date: 2013-08-30 15:50:03 Lipid storage disorders Rare diseases Nursing research Niemann–Pick disease Cyclodextrin National Institutes of Health Clinical trial Niemann-Pick disease type C Niemann-Pick disease SMPD1-associated Health Chemistry Medicine
135	Combating Rare Diseases in the 21st Century The Orphan Drug Act What is a Rare Disease? Add to Reading List Source URL: www.chi.org Language: English - Date: 2014-02-27 15:16:35 Lipid storage disorders Neurodegenerative disorders Drug discovery Epidemiology Orphan drug Orphan Drug Act Niemann–Pick disease Rare Disease Day Glycogen storage disease type II Health Medicine Rare diseases
136	New NP-C Drug Trial A clinical trial is expected to begin in the summer of 2014 using a Histone Deacetylase Inhibitors (HDACi). The goal of the Phase 1 clinical trial will be to establish the safety and tolerability of a Add to Reading List Source URL: www.parseghian.org Language: English - Date: 2013-12-11 15:45:47 Medical statistics NPC1 Clinical research Design of experiments Clinical trial Biomarker Surrogate endpoint Histone deacetylase inhibitor Niemann-Pick disease type C Medicine Health Epidemiology
137	original Article Benefits of the Orphan Drug Act for Rare Disease Treatments Boris Gites, Mona Chughtai, Jessica Chang, and M. Ian Philips A great number of people suffer from obscure and unknown diseases for which there Add to Reading List Source URL: s3.amazonaws.com Language: English - Date: 2010-05-25 15:26:09 Medicine Drug discovery Epidemiology Pharmaceuticals policy Pharmaceutical industry Orphan drug Orphan Drug Act Niemann–Pick disease Rare disease Pharmaceutical sciences Pharmacology Health
138	PDF Document Add to Reading List Source URL: www.ema.europa.eu Language: English - Date: 2013-07-21 06:20:11 Rare diseases Medicine Niemann–Pick disease Niemann-Pick disease type C Cyclodextrin Lysosomal storage disease European Medicines Agency Miglustat Orphan drug Health Chemistry Lipid storage disorders
139	PDF Document Add to Reading List Source URL: www.fda.gov Language: English Lipid storage disorders Rare diseases Pharmacology Orphan drugs Miglustat Piperidines Niemann–Pick disease Actelion Food and Drug Administration Health Medicine Pharmaceutical sciences
140	PDF Document Add to Reading List Source URL: addiandcassi.com Language: English - Date: 2010-09-07 18:10:47 Lipid storage disorders Rare diseases Niemann–Pick disease Lactones Statins Cyclodextrin NPC1 Cerebrospinal fluid Cholesterol Medicine Chemistry Health