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Rare diseases / Biology / Glycolipids / Enzymes / Galactosides / Miglustat / Lysosomal storage disease / Globoside / Ganglioside / Chemistry / Lipid storage disorders / Health


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City

Kremer / Radin / Oxon / North Brunswick / Winchester / Kulkarni / Vieira / Neville / New York / Tropak / London / L.A. / /

Company

Monsanto / McGraw-Hill / Oxford University Press / Reid S.P. / Clague A.E. / Bayer / ABC / Mehta A.S. / Matsuda / AIDS Clinical Trials Group / Cox / Tennant / Suzuki / /

Continent

Europe / /

Country

United Kingdom / Israel / United States / /

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Event

FDA Phase / Product Issues / /

Facility

National Institute of Allergy / Oxford Glycobiology Institute / University of Oxford / /

IndustryTerm

active site / excellent tools / active-site / therapy for a glycosphingolipid storage disorder / HIV therapy / treatment of Gaucher’s disease / Chemical structures / oligosaccharide processing / treatment of glycolipid lysosomal storage disorders / pharmaceutical industry interest / Treatment of Gaucher disease / membrane-associated hydrophobic site / therapy for Fabry disease / treatment of glycosphingolipid lysosomal storage diseases / therapy for type I Gaucher disease / Treatment of rare orphan diseases / action Disease therapy / storage product / HIV treatment / longer chain / therapy for storage diseases / treatment of neuronopathic diseases / treatment for lysosomal storage disorders / oral solution / /

MedicalCondition

Table I. Glycosphingolipid lysosomal storage diseases / HIV / lysosomal storage disorders / GM1 gangliosidosis ADDNJ / inherited diseases / hepatitis B / disorder / neurodegenerative gangliosidosis / GM3 deficiency / metabolic disorders / neurologic manifestations / apparent disease / inflammatory chemokine production / cancer / lysosomal disorders / parkinsonism / chronic hepadnavirus infection / Tay-Sachs disease / J. Cancer / diabetes / GM2 activator deficiency / Gaucher disease / Sandhoff disease / kidney failure / Fabry disease / sphingolipidoses / neuronopathic diseases / infantile epilepsy / Inherited Disease / Gaucher’s disease / diseases / Systemic inflammation / lysosomal storage diseases / AIDS / bovine viral diarrhea virus / inflammatory disease / deficiency / I Gaucher disease / macrophage inflammatory protein / disorders / tumours / nervous system inflammation / multidrugresistant cancer / lysosomal glycosphingolipidoses infection / human disorders / human lysosomal diseases / hepatitis C / presymptomatic Sandhoff disease / advanced HIV disease / storage diseases / peripheral neuropathy / gangliosidoses / glycolipid lysosomal storage disorders / lipid disorder / different disease phenotypes / action Disease / glycosphingolipid storage disorder / Gaucher disease type / protein misfolding disorder / neurodegenerative disorders / types II/III Gaucher disease / specific diseases / GM1 gangliosidosis / disease / T.D. Butters et al. storage disorders / enzyme inhibitor/Gaucher disease/glycosphingolipid/lysosomal storage diseases / glycosphingolipid lysosomal storage diseases / dramatic neurological dysfunction / Niemann-Pick disease / cardiovascular disease / C disease / hexosaminidase deficiency / juvenile disease / glycolipid disorders / neurodegenerative diseases / GM2 gangliosidosis / onset symptomatic epilepsy syndrome / often intractable disorders / /

MedicalTreatment

Enzyme replacement therapy / /

Organization

University of Oxford / Frances M. Platt Department of Biochemistry / National Institute of Allergy and Infectious Diseases / Oxford University / Oxford Glycobiology Institute / /

Person

van den Broek / Nat / Mellor / Allende / Perry / V / Fernandez-Guillen / Cortina-Borja / Glycobiology / Frances M. Platt / Davis / Raymond A. Dwek / Borja / Harvey / Mark Wormald / Sillence / /

Position

47R T.D. / T.D. / D.J. / Author / T.D. / and others / inhibitors 49R T.D. / 51R T.D. / infectivity 45R T.D. / biochemist / /

Product

zidovudine / Zavesca / DNJ / knockout mouse / Many / GM2 / /

ProvinceOrState

M.B. / New York / Nova Scotia / A.B. / /

Technology

X-ray / apoptosis / alpha / transplantation / UDP / pharmacokinetics / gene delivery / /

URL

http /

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