about
The cell biology of disease: lysosomal storage disorders: the cellular impact of lysosomal dysfunctionProfile of eliglustat tartrate in the management of Gaucher diseaseCyclodextrin-mediated crystallization of acid β-glucosidase in complex with amphiphilic bicyclic nojirimycin analoguesPharmacological chaperones for human -N-acetylgalactosaminidaseDeveloping therapeutic approaches for metachromatic leukodystrophyA rapid and sensitive method for measuring N-acetylglucosaminidase activity in cultured cellsFrom structural biology to designing therapy for inborn errors of metabolismThe phytoestrogen genistein modulates lysosomal metabolism and transcription factor EB (TFEB) activationGenistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic diseaseTherapeutic approaches for lysosomal storage diseasesNiemann-Pick type C pathogenesis and treatment: from statins to sugars.Pharmacological chaperone therapy for lysosomal storage diseases.Ganglioside biochemistryMiglustat improves purkinje cell survival and alters microglial phenotype in feline Niemann-Pick disease type C.Acidic Ca2+ stores in neurodegeneration.Abnormal accumulation and recycling of glycoproteins visualized in Niemann-Pick type C cells using the chemical reporter strategyβ-Glucosidase 2 (GBA2) activity and imino sugar pharmacology.Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Pharmacological chaperone therapy for Gaucher disease: a patent review.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Blood-brain barrier structure and function and the challenges for CNS drug delivery.Lysosomal storage diseases--the horizon expands.Sphingolipid lysosomal storage disorders.Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.N-Alkyl-, 1-C-Alkyl-, and 5-C-Alkyl-1,5-dideoxy-1,5-imino-(L)-ribitols as Galactosidase Inhibitors.Gaucher disease: Progress and ongoing challenges.Lipid glycosylation: a primer for histochemists and cell biologists.The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblastsStructural basis of pharmacological chaperoning for human β-galactosidase.Pharmacologic Treatment Assigned for Niemann Pick Type C1 Disease Partly Changes Behavioral Traits in Wild-Type Mice.Human pluripotent stem cell models of cardiac disease: from mechanisms to therapies.Increased Regenerative Capacity of the Olfactory Epithelium in Niemann-Pick Disease Type C1.Lysosomal Storage Disorders and Malignancy.A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.Ambroxol as a pharmacological chaperone for mutant glucocerebrosidase.A systematic investigation of iminosugar click clusters as pharmacological chaperones for the treatment of Gaucher disease.Pharmacological Chaperones as Potential Therapeutic Strategies for Misfolded Mutant Vasopressin Receptors.
P2860
Q24603159-4FE578CD-5469-495C-83E5-FDF57746066AQ26774416-DD9CB985-378C-49E3-B443-0EE6987EAA63Q27667205-401A2D1B-F218-45FA-B21A-BE8AD22724CEQ27674346-3238C66C-8CA7-4455-B279-0F053AB56C6DQ28297086-594EB22C-2C1E-49ED-9482-5B13E7CE039FQ28534395-162203F4-4510-4F45-ABBC-C47CA44C72C4Q28828914-44180C5C-0BB9-46C4-A4A6-6B765E9B232AQ33761283-190B8981-5CCD-4DD6-9C52-76DCD71639FDQ33769790-4433A9AB-6E75-40DC-B902-2930AF33ED3EQ34311420-93CB93E4-EFF0-4760-B24A-1E6E62E3E6E5Q34632493-68FF8F8A-54B6-4A15-8B64-FB7B78928092Q35214286-64DBB618-0BD0-41F3-AF10-6B0C21E7027CQ35361488-BFE94476-8E00-4353-8B8B-EADAE6A4CA1DQ35960437-12B5DCDB-448C-410B-9FB6-29E0C42C7BD2Q36395510-BC75DC4F-05AE-4A86-8996-04420C7376EBQ36950303-8513A57D-63EB-4E66-824B-4F8C032CDDEAQ37151058-AA4BC715-A415-4B79-9839-5B938FCEC220Q37668227-E164B706-469C-4CBF-8A6E-08602F42B7C7Q37672410-75D3CC73-F81B-4D84-ABBB-1E9E2312DB3BQ37860332-82B8FED5-ACDF-40DD-900D-466D450A7E37Q37876354-AE56AB83-859F-4B59-8366-8CD7AB32CC8EQ38100970-17BE68DB-B45B-4DF3-A5DA-3AB823EB70DBQ38128478-324F373F-4C96-45DD-8269-BA780F14B010Q38217766-36DC2C43-19EE-4CAE-8268-CA725236EF13Q38424066-984EF5EC-8D86-42CB-83FB-058E9459EA7BQ38684097-B820C7D7-D115-45B2-9FBB-974B74D16049Q38797648-E3243870-EBE7-4C21-A48D-C8ED045FCD1AQ38813807-E79DF1BF-EC98-4933-B606-8E0CF49429F1Q39028972-91FF2C86-6FDB-4378-A9C2-948CB2BDCA25Q39045114-5A5B17BD-6FBF-481C-B3B0-BA10F4FE2769Q39508157-FCED00EB-B5B9-4962-B8F6-5A883719EE24Q39885525-0C438EFE-0685-4893-8236-9ACABB6D1DE5Q40479802-D7088299-09B9-4DB0-9D04-D320633D0F91Q41664792-E839DF60-5984-4FC0-B5DB-785ACDCB5F91Q41953682-15DB47FF-4902-4701-9F59-D7354087BDC3Q41971408-E9BCF72C-3E3D-4FDA-9A58-288CD0EC6CA6Q42226747-2EE2C5F2-E74E-4708-85BA-FE3B4ECF0565Q42763507-4C33519F-AD45-443F-AAEF-8A7D67FAEC6DQ45716125-CBB015DF-AC1B-4EC2-AD81-3540DD3FB734Q47757944-9B2178EE-B458-43D4-BB50-DCE4D76CEFBB
P2860
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on April 2008
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Substrate reduction therapy.
@en
Substrate reduction therapy.
@nl
type
label
Substrate reduction therapy.
@en
Substrate reduction therapy.
@nl
prefLabel
Substrate reduction therapy.
@en
Substrate reduction therapy.
@nl
P2860
P1433
P1476
Substrate reduction therapy.
@en
P2093
Mylvaganam Jeyakumar
P2860
P356
10.1111/J.1651-2227.2008.00656.X
P407
P577
2008-04-01T00:00:00Z