Long-term effect of imiglucerase in Latin American children with Gaucher disease type 1: lessons from the International Collaborative Gaucher Group Gaucher Registry

Conclusion Continuous and long-term treatment with imiglucerase improves hematological, visceral and skeletal manifestations of Gaucher disease type 1. Trial registration NCT00358943.
Source: BMC Hematology - Category: Hematology Source Type: research