A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. - Wikidata - wikimedia - TriplyDB

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.

http://www.wikidata.org/entity/Q33389419

about

Glucocerebrosidase is shaking up the synucleinopathies Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders Profile of eliglustat tartrate in the management of Gaucher disease Eliglustat tartrate for the treatment of adults with type 1 Gaucher disease Podocyte pathology and nephropathy - sphingolipids in glomerular diseases On ceramides, other sphingolipids and impaired glucose homeostasis Binding of 3,4,5,6-Tetrahydroxyazepanes to the Acid-β-glucosidase Active Site: Implications for Pharmacological Chaperone Design for Gaucher Disease Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy Developing novel chemical entities for the treatment of lysosomal storage disorders: an academic perspective Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.Revised recommendations for the management of Gaucher disease in children.The design and clinical development of inhibitors of glycosphingolipid synthesis: will invention be the mother of necessity?Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse.Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat.Development of targeted therapies for Parkinson's disease and related synucleinopathies ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.Therapeutic approaches for lysosomal storage diseases Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage.Gaucher's disease and cancer: a sphingolipid perspective Gaucher disease and its treatment options.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease Gaucher disease: clinical profile and therapeutic developments.A reappraisal of Gaucher disease-diagnosis and disease management algorithms.Biomarkers in the diagnosis of lysosomal storage disorders: proteins, lipids, and inhibodies.Scientific literature on monosialoganglioside in the Science Citation Index-Expanded: A bibliometric analysis of articles from 1942 to 2011 by each decade Reducing selection bias in case-control studies from rare disease registries.Ubiquitous transgene expression of the glucosylceramide-synthesizing enzyme accelerates glucosylceramide accumulation and storage cells in a Gaucher disease mouse model.Dissociation of ERK signalling inhibition from the anti-amyloidogenic action of synthetic ceramide analogues Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain.AMP-activated Protein Kinase Suppresses Biosynthesis of Glucosylceramide by Reducing Intracellular Sugar Nucleotides.Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options.

P2860

Q24337392-BE230763-1F90-4C50-8C6A-38AAB411C3FE Q26744230-EDA8A441-CF84-4A23-A46D-6855FFEDB0AC Q26774416-1CA9B87A-858E-4972-82D8-373E939DFFD1 Q26783393-88140B02-00F3-41CF-A8E3-5F9E435B921C Q26852463-D4E927D4-78D8-4759-B8B2-1C2831F404BC Q27021446-010B421B-D83A-4A40-841D-DF549C853192 Q27675348-CD287400-6830-4AD5-BC1B-CB69501730BE Q28067032-3E861D7F-C04E-4550-8FD1-709669AEF130 Q28086909-50DF58FA-4B07-4990-82F1-8C894506E612 Q28478879-3B885901-4DC1-41B2-A5CA-4879D532DE8D Q28478911-7B2205C4-066B-4C82-AF34-F675DFF0713A Q28748170-D5CFBF98-373D-45FF-B62E-FA05B8BED07C Q30235844-F338A010-D5CC-4CEC-AC19-00B2BAB5627F Q33391428-7FC562ED-5622-42B9-A574-21302C304FC5 Q33402191-EEDAF006-BA7E-4F89-9FEB-9C6C45579560 Q33409263-1AD3F612-B469-4A33-8265-56FC4E79E314 Q33420506-E9E00098-1895-40C2-A0A6-ADD2873D2B4E Q33435875-2CB9F398-CDF0-43A6-A8D4-E58D3716605D Q33615625-DD9AE100-6B58-40C3-B0A5-1B209AABA06E Q33886270-74C9BE39-D1EE-470B-97A7-7B33BCEEBE96 Q34079226-E4B8ABE2-09C7-4BB5-A0A4-C75AF2032C2C Q34238616-091ED890-3D41-497F-9E3E-8D7103E445FF Q34273177-49E7FC1E-ADCE-4548-AB3F-5B0054F25F1C Q34311420-7F0D359E-178F-4F7F-8074-4EE914FA15D7 Q34320470-C87F2C83-62D8-4F8B-B1B4-136743FDD496 Q34333982-B4577313-B54E-4D61-8A33-5A9228F99DE3 Q34386658-0B7C6E60-FE01-4898-ADD6-136CCC462215 Q34440333-681E4726-EAE6-4A03-B143-6F5413204EC4 Q34443517-CA8DA0B3-EFE9-41C5-B969-34FC0D661814 Q34683786-3D9ABE22-8195-492D-A87F-138315BDA1E8 Q35028532-D4F92A70-126C-4FA9-ABE0-67F1FBDB575F Q35160719-156986F7-F08C-4D53-BBDA-95ED7EC91711 Q35439561-69299454-AD60-4CE6-A990-31140B8B5772 Q35536435-65EED9BD-8AEF-44AA-AB93-47085C114492 Q35676462-F7238F16-3F3B-407C-99FB-079029A1605D Q35715564-F7E3489C-6F2A-40CD-9360-AB6720918F6F Q35859585-EB914E9F-5A09-46BC-BFA8-ACF47E0EE784 Q36725221-A5B3F611-F04B-4AE6-A444-0619ADAB1704 Q37043500-C6EA8933-D699-4194-A878-51F9DF2E2195 Q37167690-C689DC36-9472-480F-AF80-D5A6DB93C3EC

P2860

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1.

http://www.wikidata.org/entity/Q33389419

description

2010 nî lūn-bûn

@nan

2010 թուականի Մայիսին հրատարակուած գիտական յօդուած

@hyw

2010 թվականի մայիսին հրատարակված գիտական հոդված

@hy

2010年の論文

@ja

2010年学术文章

@wuu

2010年学术文章

@zh-cn

2010年学术文章

@zh-hans

2010年学术文章

@zh-my

2010年学术文章

@zh-sg

2010年學術文章

@yue

name

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@ast

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@en

type

label

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@ast

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@en

prefLabel

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@ast

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@en

P1433

Q33389419-0AA39D7C-17C5-41CF-8BF8-22B60350201F

P1476

Q33389419-9F30415F-9C4C-4C66-B17A-9EA92AA3B9E7

P2093

Q33389419-010F03FE-FC98-4B56-81E8-04C1197E6557

Q33389419-0A9195EB-EFB0-4C2F-AC7B-E31B2A3E5354

Q33389419-1A798DBD-C546-4AD2-A5B8-443B2D62DA70

Q33389419-248FACCE-50AC-4C43-BB98-F4433448E7D7

Q33389419-2865FD00-EF7F-4451-816C-87375DE7D50B

Q33389419-406C7517-BD9A-4723-BF8D-1838DA79D2CB

Q33389419-4A0E9899-B141-43F4-B8F7-E5A8161E2EA6

Q33389419-4BCC67F1-D002-4188-9BF1-0441530350A7

Q33389419-6B80F9C7-19D2-4943-9F85-A0D250D86CA9

Q33389419-877AFD3E-B1C6-4C1E-ACC0-B304E20C54C4

P2860

Q33389419-3C68561D-4CC3-47BC-9A1B-46A96EBE6ED5

Q33389419-59BA9DFF-3D43-4940-85A6-1DF86AA76DF4

Q33389419-699F495B-3FA0-4304-A49C-6DC07DA74028

Q33389419-850E444A-BF35-4839-8E1D-55779FD7203B

Q33389419-9085DC7D-7486-46A7-8A04-23FDF0D27475

Q33389419-990B0A42-45D0-41D3-9FC7-F79C8C6DD5E1

Q33389419-CC65825E-CBA8-4E59-9D9B-1DF0802B6453

Q33389419-D0FFCE95-0F49-4625-B777-3503662F7DD6

Q33389419-D8C43764-1870-4F9F-BB67-E5B575A06229

Q33389419-F78294DC-44B7-44DA-87B2-1131BBB01E37

P304

Q33389419-25DD7F59-6144-4B9D-8E93-A0318B9DCABD

P31

Q33389419-AC0477BD-1D82-4DDA-8675-39AC3C2A09C2

P356

Q33389419-81C2DF1C-FA86-43EB-A17A-E6299002D3C9

P407

Q33389419-5314F8CD-BEA3-4103-A4D6-4F28F946C78B

P433

Q33389419-D61C1189-0983-4A51-912C-90F566679B4C

P478

Q33389419-D3383C3C-75FF-4C19-918F-10BB3AFC6DF2

P577

Q33389419-09FE49B0-43CC-4027-B9CD-877722DFFB18

P5875

Q33389419-05C8539D-A1C1-4661-8F8A-1EF36FCBD122

P698

Q33389419-B2216ABB-10EC-4272-8A60-18F7ED8C4536

P921

Q33389419-08BFEC78-544A-429B-BBB9-9F25316BD61F

Q33389419-852F3096-7F15-4BDC-83FA-AA449CC8795B

Q33389419-9C9C3242-AF81-4EA1-95C6-F8F44D19BBC1

P932

Q33389419-7AC4C886-A45D-4F4E-A45E-AE4EDE6F1399

P356

BLOOD-2010-03-273151

P1433

P1476

A phase 2 study of eliglustat ...... py for Gaucher disease type 1.

@en

P2093

Ana Cristina Puga

http://www.w3.org/2001/XMLSchema#string

Daniel I Rosenthal

http://www.w3.org/2001/XMLSchema#string

Elena Lukina

http://www.w3.org/2001/XMLSchema#string

Elsa Avila Arreguin

http://www.w3.org/2001/XMLSchema#string

Gregory M Pastores

http://www.w3.org/2001/XMLSchema#string

Hanna Rosenbaum

http://www.w3.org/2001/XMLSchema#string

M Judith Peterschmitt

http://www.w3.org/2001/XMLSchema#string

Marcelo Iastrebner

http://www.w3.org/2001/XMLSchema#string

Marta Dragosky

http://www.w3.org/2001/XMLSchema#string

Maryam Banikazemi

http://www.w3.org/2001/XMLSchema#string

P2860

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry.

Therapeutic goals in the treatment of Gaucher disease.

Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease

Official Positions of the International Society for Clinical Densitometry and executive summary of the 2007 ISCD Position Development Conference.

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1.

Gaucher disease: a model disorder for biomarker discovery.

Type I Gaucher disease, a glycosphingolipid storage disorder, is associated with insulin resistance.

Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies.

Gaucher disease in Colombia: mutation identification and comparison to other Hispanic populations.

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

P304

893-899

http://www.w3.org/2001/XMLSchema#string

P31

scholarly article

P356

10.1182/BLOOD-2010-03-273151

http://www.w3.org/2001/XMLSchema#string

P407

P433

6

http://www.w3.org/2001/XMLSchema#string

P478

116

http://www.w3.org/2001/XMLSchema#string

P577

2010-05-03T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P5875

44570535

http://www.w3.org/2001/XMLSchema#string

P698

20439622

http://www.w3.org/2001/XMLSchema#string

P921

eliglustat tartrate

P932

2924227

http://www.w3.org/2001/XMLSchema#string