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J-ACT: Ambroxol chaperone therapy in neuronopathic Gaucher disease
jRCTs061190017Active, not recruiting
- Sponsor
- JCR Pharmaceuticals
- Start
- Nov 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label trial of ambroxol in Gaucher disease (GD) (2 cohorts), sponsored by JCR Pharmaceuticals. Primary endpoint: Glucosylsphingosine concentrations in cerebrospinal fluid. Started Nov 2019; status: active, not recruiting.
02
Drugs
ambroxol
03
Conditions
Gaucher disease (GD)
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