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Observational Study of Plasma Lyso-Gb1 and Treatment Outcomes in Japanese Gaucher Disease Patients
jRCT1031200137Completed
- Sponsor
- Takeda
- Start
- Oct 2020
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Open-label, single-arm trial of velaglucerase alfa in Gaucher disease (GD), sponsored by Takeda. Primary endpoint: Correlation of lyso-Gb1 with number of treatment targets achieved. Started Oct 2020; status: completed.
02
Drugs
velaglucerase alfa
03
Conditions
Gaucher disease (GD)
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