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Ambroxol in neuronopathic and Parkinsonian Gaucher disease
2021-006224-40Phase 2Completed
- Sponsor
- Children's Memorial Health Institute
- Start
- May 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ambroxol in Gaucher disease (GD) and Gaucher disease type 3, sponsored by Children's Memorial Health Institute. Primary endpoint: Neurological clinical status. Started May 2022; status: completed.
02
Drugs
ambroxol
03
Conditions
Gaucher disease (GD)Gaucher disease type 3
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