Safety and Efficacy of Ambroxol Therapy in Polish Patients with Gaucher Disease
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.
Authors
- Małgorzata Syczewska (ORCID: https://orcid.org/0000-0002-8872-6476)
- Mirosław Kwaśniewski
- Karolina Chwiałkowska (ORCID: https://orcid.org/0000-0001-8053-8959)
- Patryk Lipiński (ORCID: https://orcid.org/0000-0002-1849-8375)
- Joanna Godzień (ORCID: https://orcid.org/0000-0002-9477-057X)
- Paulina Szymańska-Rożek (ORCID: https://orcid.org/0000-0002-2527-5081)
- Aleksandra Jezela-Stanek
- Anna Tylki-Szymanska
- Michał Ciborowski
- Urszula Korotko
- Dariusz Rokicki
- Magdalena Niemira
Institutions
- Medical University of Białystok (PL)
- Medical University of Warsaw (PL)
- Netherlands Metabolomics Centre (NL)
- Children's Memorial Health Institute (PL)
- Genomics Medicine (Ireland) (IE)
- Postgraduate School of Molecular Medicine (PL)
- University of Warsaw (PL)
Publication Details
- Journal
- Life
- Published
- 2026-03-16
- DOI
- https://doi.org/10.3390/life16030485
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00