Safety and Efficacy of Ambroxol Therapy in Polish Patients with Gaucher Disease

20260 citationsJournal Articlegold Open Access

Authors

Patryk Lipiński · Postgraduate School of Molecular Medicine

Dariusz Rokicki · Children's Memorial Health Institute

Karolina Chwiałkowska · Postgraduate School of Molecular Medicine

Michał Ciborowski · Medical University of Białystok

Joanna Godzień · Netherlands Metabolomics Centre

Aleksandra Jezela-Stanek

Urszula Korotko · Medical University of Warsaw

Abstract

In patients with type 3 GD receiving stable ERT, ambroxol demonstrated beneficial effects on neurological symptom expression. Some improvement was observed in biomarkers; the activity of chitotrosidase and concentration of lyso-Gl1 decreased. These findings support the therapeutic potential of ambroxol as an adjunctive treatment for neuronopathic Gaucher disease.

Topics & Keywords

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Trypanosoma species research and implications

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Life

Volume 16, Issue 3, pp. 485-485

DOI: 10.3390/life16030485

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