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Rare diseases / Biology / Neurodegenerative disorders / Niemann–Pick disease / Epidemiology / Nursing research / Niemann-Pick disease / type C / Cyclodextrin / NPC1 / Health / Medicine / Lipid storage disorders


Spring[removed]Volume 20 No 1 CONTENTS Chairman’s Chat
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Document Date: 2013-06-25 13:27:58

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City

Oxford / Manchester / Concord / New York City / Dublin / Urbana / Cardiff / Frankfurt / /

Company

Willink Laboratory / NP Suisse / Monmouth Dora Bryan OBE Rt. / Johnson and Johnson / Actelion Pharmaceuticals / INPDA Executive Committee 6 Niemann-Pick Disease Group / Salford Royal Foundation Trust / Niemann-Pick Disease Group / HP / NCATS / /

Continent

Europe / North America / /

Country

France / United Kingdom / United States / /

Currency

GBP / USD / EUR / /

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Event

FDA Phase / Product Issues / Employment Change / /

Facility

Cardiff University / The University of Oxford / Willink Laboratory / Eunice Kennedy Shriver National Institute of Child Health / NIH’s National Center / University of Notre Dame / NIH’s National Human Genome Research Institute / Washington University / National Institute of Neurological Disorders / Hospital Oxford Road / NIH Clinical Center / University of Oxford / Manchester Hospital / Manchester Airport / NIH’s National Institute of Neurological Disorders / Thistle Hotel / University of Pennsylvania / /

IndustryTerm

repair solutions / treatment for a devastating disease / therapies for rare diseases / treatment of genetic diseases / oxysterol diagnostic tool / treatment for Niemann-Pick disease / biotech/pharmaceutical / Therapeutics for Rare and Neglected Diseases / diagnostic tool / produce preliminary data enabling applications / /

MedicalCondition

Type C. hereditary inclusion body myopathy / rare diseases / Lysosomal Storage Disorders / rare and fatal genetic disease / difficult diagnosable disease / Most rare diseases / lipoprotein lipase deficiency / Niemann-Pick diseases / orphan disease / C (NPC) disease / rare neurological lysosomal storage diseases / genetic diseases / Rare Disorders / diseases / total shock / NPC1 Disease / lysosomal diseases / little nausea / common neurodegenerative and aging disorders / lethal neurodegenerative disease / chronic lymphocytic leukemia / rare inherited genetic disease / rare disorder / mechanisms rare diseases / NPC disease / disease / common disorders / Neglected Diseases / NiemannPick disease / rare disease / similar inherited rare genetic diseases / Niemann-Pick disease / neurological disease / C disease / rare genetic disease / rare and neglected diseases / sickle cell disease / tropical diseases / neurological diseases / genetic disorders / /

MedicalTreatment

surgery / gene therapy / Enzyme Replacement Therapy / /

Organization

NIH’s National Human Genome Research Institute / Willink Unit / Washington University in St. Louis School of Medicine / NIH Clinical Center / NIH’s National Institute of Neurological Disorders and Stroke / TRND / National Niemann-Pick Disease Foundation / University of Oxford / Centerville Elementary School / NNPDF Central Office / German NP / Genetic Alliance / European Commission / Management Committee / University of Pennsylvania / Philadelphia / CLIMB / EURORDIS / Ara Parshegian Medical Research Foundation / European Union / Department of Health / NHS / Committee for Orphan Medicinal Products / Award Committee / Manchester Hospital / National Institute of Health / Food and Drug Administration / Cardiff University / International Niemann Pick Disease Alliance / Human Development / Specialised Healthcare Alliance / CONTENTS / NIH’s National Center for Advancing Translational Sciences / Board of Trustees / Eunice Kennedy Shriver National Institute of Child Health and Human Development / Eunice Kennedy Shriver National Institute of Child Health / Albert Einstein School of Medicine / NCATS office of Communications / University of Notre Dame / Baltimore Orioles / /

Person

Janice Brooks / Dillon Papier / Christa Parseghian / Richard Rogerson Jim Green / Forbes Porter / Brian Roberts / Christopher P. Austin / Michael / Jackie Imrie / Guy Johnston Nicholas Mathias / Simon Jones / Denise Nolan Keith Andrews / Richard Brooks / Martin Rossor / Nuria Carrillo / Christoph Poincilit / Bassam / Peter G. Pentchev / Elizabeth Davenport / Innovation / Washington Tyne / Forbes D. Porter / Bill Owen / Laura Bell / Dominic Walker / Fran Platt / Helen Carter / Marcia Parseghian / John McKew / Toni Mathieson / Sue Lowe / David Roberts / /

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Position

Executive Director / senior investigator / Rt / chief / acting director / Trustee and Treasurer / Information Officer / Professor / 2nd baseman / staff physician / clinical director / Director Niemann-Pick Disease Group / Director Email / Medical Advisor / Clinical Nurse Specialist Department of Metabolic Medicine / OGS Bishop / Chairman / Chairman / the Medical Advisor / the Research / director / favorite player / Commissioner for Research / Clinical Nurse Specialist / Nurse / uk Chairman / Porter / Families Officer / European Commissioner for Research / Our Clinical Nurse Specialist / Our Families Officer / chief of the Therapeutic Development Branch and director / /

Product

Investigational New Drug / Cyclodextrin / NPC / proteins / /

ProvinceOrState

Indiana / Maryland / Illinois / Pennsylvania / /

RadioStation

CBE / /

Technology

Neuroscience / stem cells / html / Human Genome / gene therapy / /

URL

http /

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