Safety and Efficacy of Ambroxol Therapy in Polish Patients with Gaucher Disease - PubMed
6 hours ago
- #Gaucher Disease
- #Neurological Symptoms
- #Ambroxol Therapy
- Ambroxol therapy was evaluated in 13 Polish patients with type 3 Gaucher disease, who were L444P homozygotes, over one year at a dose of 10 mg/kg/day.
- The study found that ambroxol led to a reduction or complete resolution of selected neurological symptoms in several patients.
- Biomarker improvements were observed, including decreased activity of chitotriosidase and concentration of glucosylsphingosine (Lyso-GL1).
- Ambroxol showed beneficial effects on neurological symptoms in type 3 Gaucher disease patients already on stable enzyme replacement therapy (ERT).
- The results support ambroxol's potential as an adjunctive treatment for neuronopathic forms of Gaucher disease, particularly due to its ability to cross the blood-brain barrier.