Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.
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Enzyme replacement and substrate reduction therapy for Gaucher diseaseLess Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersProfile of eliglustat tartrate in the management of Gaucher diseaseEliglustat tartrate for the treatment of adults with type 1 Gaucher diseaseDeveloping novel chemical entities for the treatment of lysosomal storage disorders: an academic perspectiveA Review of Gaucher Disease Pathophysiology, Clinical Presentation and TreatmentsHematological manifestations and complications of Gaucher disease.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 trialTreatment of profound thrombocytopenia in a patient with Gaucher disease type 1: Is there a role for substrate reduction therapy.Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapyOnce again, rare diseases provide a spotlight.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry DiseaseElevation of glycoprotein nonmetastatic melanoma protein B in type 1 Gaucher disease patients and mouse modelsClonal Immunoglobulin against Lysolipids in the Origin of MyelomaBiochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patientsA new framework for evaluating the health impacts of treatment for Gaucher disease type 1.Targeting Wolman Disease and Cholesteryl Ester Storage Disease: Disease Pathogenesis and Therapeutic Development.Eliglustat: A Review in Gaucher Disease Type 1.Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies.Gaucher disease: Progress and ongoing challenges.Type II NKT Cells and Their Emerging Role in Health and Disease.Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trialHematopoietic stem cell transplantation for Gaucher disease.Response to Letter to the Editor on "Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options".Letter to the Editor on "Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options".Antigen-mediated regulation in monoclonal gammopathies and myeloma.Pharmacotherapy of Gaucher Disease: Current and Future Options.Targeting Glycosphingolipid Metabolism to Treat Kidney Disease.A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease
P2860
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P2860
Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.
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2015 nî lūn-bûn
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2015 թուականի Փետրուարին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի փետրվարին հրատարակված գիտական հոդված
@hy
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@ast
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@en
type
label
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@ast
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@en
prefLabel
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@ast
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@en
P2093
P2860
P356
P1476
Effect of oral eliglustat on s ...... AGE randomized clinical trial.
@en
P2093
Andres Ortega
Atul Mehta
Dominick Amato
Elena Lukina
Evgueniy Hadjiev
Gregory Pastores
Hadhami Ben Turkia
Hagit Baris
Jennifer Angell
Leorah Ross
P2860
P304
P356
10.1001/JAMA.2015.459
P407
P577
2015-02-01T00:00:00Z