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Velaglucerase alfa enzyme replacement in children and adolescents with type 3 Gaucher disease
2012-003427-38Phase 2
- Sponsor
- Takeda
- Start
- Apr 2016
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of velaglucerase alfa in Gaucher disease type 3 and Gaucher disease type 1 (GD1), sponsored by Takeda. Primary endpoint: Change From Baseline to 12 Months in Hemoglobin Concentration.
02
Drugs
velaglucerase alfa
03
Conditions
Gaucher disease type 3Gaucher disease type 1 (GD1)
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