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World Data on Ambroxol for Patients With GD and GBA Related PD

RecruitingN/A

Sponsor: Shaare Zedek Medical Center

Conditions: Gaucher Disease, Parkinson Disease, GBA Gene Mutation

Countries: Israel

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).

Eligibility overview

Sex: ALL

Age: — to 100 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

* patients with Gaucher disease type 1,2 or 3(a,b,c).
* patients with GBA-related Parkinson disease.

Exclusion Criteria:

* None.

Locations (3)

Jerusalem, Please Select..., Israel
Jerusalem, Israel
Jerusalem, Israel