Early initiation of high-dose oral ambroxol in combination with enzyme replacement therapy in a neuropathic Gaucher infant-Reference-Cited by-同舟云学术

Early initiation of high-dose oral ambroxol in combination with enzyme replacement therapy in a neuropathic Gaucher infant

Published:2020-03 Issue: Volume:81 Page:102402
ISSN:1079-9796
Container-title:Blood Cells, Molecules, and Diseases
language:en
Short-container-title:Blood Cells, Molecules, and Diseases

Author:

Chu Shao-Yin,Chien Chun-Ching,Hwu Wuh-Liang,Wang Pen-Jung,Chien Yin-Hsiu

Publisher

Elsevier BV

Subject

Cell Biology,Hematology,Molecular Biology,Molecular Medicine

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3. Individualized screening for chaperone activity in Gaucher disease using multiple patient derived primary cell lines;Ivanova;Am. J. Transl. Res.,2018

4. Performance of the four-Plex tandem mass spectrometry lysosomal storage disease newborn screening test: the necessity of adding a 2nd tier test for Pompe disease;Chiang;International Journal of Neonatal Screening,2018

5. Mutation analysis of Gaucher disease patients in Taiwan: high prevalence of the RecNciI and L444P mutations;Wan;Blood Cells Mol. Dis.,2006

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1. Clinical and preclinical insights into high-dose ambroxol therapy for Gaucher disease type 2 and 3: A comprehensive systematic review;Molecular Genetics and Metabolism;2024-09

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3. Rapid and long‐lasting efficacy of high‐dose ambroxol therapy for neuronopathic Gaucher disease: A case report and literature review;Molecular Genetics & Genomic Medicine;2024-03-30

4. Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies;Frontiers in Pharmacology;2024-02-13

5. The use of Ambroxol for the treatment of Gaucher disease: A systematic review;eJHaem;2024-01-30