Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease-Reference-Cited by-同舟云学术

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Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease

Published:2021-12 Issue: Volume:29 Page:100798
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Kleytman Nathaniel,Ruan Jiapeng,Ruan Audrey,Zhang Bailin,Murugesan Vagishwari,Lin Haiqun,Guo Lilu,Klinger Katherine,Mistry Pramod K.

Funder

National Institutes of Health

Sanofi Genzyme

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference31 articles.

1. Gaucher disease: basic and translational science needs for more complete therapy and management;Grabowski;Mol. Genet. Metab.,2021

2. The Online Metabolic and Molecular Bases of Inherited Disease,2010

3. Replacement therapy for inherited enzyme deficiency–macrophage-targeted glucocerebrosidase for Gaucher’s disease;Barton;N. Engl. J. Med.,1991

4. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment;Weinreb;Mol. Genet. Metab,2021

5. Therapeutic delivery of proteins to macrophages: implications for treatment of Gaucher’s disease;Mistry;Lancet,1996

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1. Gaucher Disease: A Glance from a Medicinal Chemistry Perspective;ChemMedChem;2024-03-08

2. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach;International Journal of Biological Sciences;2024

3. Real life data: follow-up assessment on Spanish Gaucher disease patients treated with eliglustat. TRAZELGA project;Orphanet Journal of Rare Diseases;2023-12-15

4. A new multiplex analysis of glucosylsphingosine and globotriaosylsphingosine in dried blood spots by tandem mass spectrometry;Molecular Genetics and Metabolism Reports;2023-12

5. Effectiveness and Safety of Eliglustat Treatment in Gaucher Disease: Real-life Unicentric Experience;Clinical Therapeutics;2023-11

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