Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: OBSERVATIONAL
Official Title: IIR REGISTRY for the Collection of Real World Data on the Safety and Efficacy of Ambroxol for Patients With Gaucher Disease or GBA Carriers With Parkinson Disease
Brief Summary: Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine.
In 2009 it was found to also act as a pharmacological chaperone (PC) for mutant glucocerebrosidase, albeit in a several-fold higher dose.
Unfortunately, due to its low cost, there have been no pharma-driven clinical trials to establish the use of ambroxol.
Thus, data are needed on the safety and efficacy of ambroxol for patients with Gaucher disease (GD).