Condition: Gaucher Disease · Parkinson Disease · GBA Gene Mutation · Sponsor: Shaare Zedek Medical Center
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).
This description comes directly from the study's public registry record.
Majdolen Istaiti, BS.C · +972-2-5645681 · joleenist@szmc.org.il
Ari Zimran, MD · +972-2-5645681 · azimran@gmail.com
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| Shaare Zedek Medical Center | Jerusalem, Please Select..., Israel | Recruiting |
| Shaare Zedek Medical Center | Jerusalem, Israel | Recruiting |
| Shaare Zedek Medical Center | Jerusalem, Israel | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04388969