Study of Velaglucerase Beta ERT in Children With Gaucher Disease

Authors
Category Primary study
Registry of TrialsClinicalTrials.gov
Year 2026
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
Epistemonikos ID: 4f30e7053560078d2d55ba14e7c3ad9ae422d370
First added on: Sep 30, 2026