Patent
Pharmaceutically active piperidine derivatives
Butters Terence D,Raymond A. Dwek,George William John Fleet,Michael Glen Orchard,Frances M. Platt +4 more
TLDR
In this paper, a compound of formula (I) wherein R represents various substituent groups, is used as an inhibitor of glucosylceramide synthase and is shown to be useful as a deterministic inhibitor.Abstract:
Compounds of formula (I) wherein R represents various substituent groups, are useful as inhibitors of glucosylceramide synthase.read more
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Patent
Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
David Meeker,Seng H. Cheng +1 more
TL;DR: In this article, a combination of enzyme replacement therapy, gene therapy, and small molecule therapy was used for the treatment of lysosomal storage diseases. But, their method was not suitable for the use of small molecules.
Patent
Deoxynojirimycin analogues and their uses as glucosylceramidase inhibitors
TL;DR: In this paper, a new class of deoxynojirimycin analogues, or pharmaceutically acceptable salts thereof which can suitably be used for the treatment of a disease selected from the group consisting of insulin resistance, Gauger disease, inflammatory diseases, hyperpigmentation and/or inflammatory skin conditions, overweight and obesity, lysosomal storage disorders, fungal diseases, melanoma and other tumors, and microbacterial infections.
Patent
2-acylaminopropoanol-type glucosylceramide synthase inhibitors
Craig Siegel,Cecilia M. Bastos,David J. Harris,Angeles Dios,Edward R. Lee,Richard Silva,Lisa M. Cuff,Mikaela Levine,Cassandra Celatka,Frederic Vinick,Thomas H. Jozefiak,Yibin Xiang,John L. Kane,Junkai Liao +13 more
TL;DR: A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof as mentioned in this paper. And a pharmaceutical composition comprises a compound represented by structural formula (I) or a pharmaceutical salt thereof.
Patent
Methods of treating diabetes mellitus
TL;DR: In this paper, the authors described methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject, which they described as a "minimally effective" method.
Patent
Epitope reduction therapy
TL;DR: In this paper, the use of an inhibitor of glycolipid biosynthesis in the manufacture of a medicament for the treatment of a GML-mediated autoimmune disease was described.
References
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Book
Protective groups in organic synthesis
TL;DR: In this article, the most useful groups for the hydroxyl, amino, carboxyl, carbonyl, and sulfhydryl groups are discussed and the chemistry of the classes of these groups, as well as that of individual groups within the class using structures, equations and references.
Journal ArticleDOI
Functional rafts in cell membranes
Kai Simons,Elina Ikonen +1 more
TL;DR: A new aspect of cell membrane structure is presented, based on the dynamic clustering of sphingolipids and cholesterol to form rafts that move within the fluid bilayer that function as platforms for the attachment of proteins when membranes are moved around inside the cell and during signal transduction.
Book
A Laboratory manual
TL;DR: To develop a program to print the barcodes using two commonly uses command sets and hence evaluates their ease of use for such applications, students should be able to program dot matrix printers, by manipulating bit level information and ink jet printers using page description language, such as PCL.
Journal ArticleDOI
Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
Timothy M. Cox,Robin H. Lachmann,C. E. M. Hollak,Johannes M. F. G. Aerts,S. van Weely,M. Hrebicek,Frances M. Platt,Terry D. Butters,Raymond A. Dwek,C. Moyses,I. Gow,Deborah Elstein,Ari Zimran +12 more
TL;DR: Reducing the rate of substrate formation by OGT 918 improves key clinical features of non-neuronopathic Gaucher's disease and justifies further trials in this and other glycosphingolipid storage disorders.
Journal ArticleDOI
GM1 ganglioside-bound amyloid beta-protein (A beta): a possible form of preamyloid in Alzheimer's disease.
TL;DR: The presence of membrane–bound Aβ that tightly binds CM1 ganglioside suggests that this novel Aβ species, rather than secreted Aβ, may act as a ‘seed’ for amyloid and intracellular abnormalities in the membrane recycling already exist at the stage of amyloidsogenesis.