Aisha I Abdullah PhD,  —

A bone marrow transplant successfully reduced the severe lung involvement that developed in a 5-year-old boy with Gaucher disease who was receiving enzyme replacement therapy (ERT), a study reported. The boy was described in a previous report as the youngest patient…

An experimental gene therapy known as AVR-RD-02 has received orphan drug designation from the European Commission for the treatment of Gaucher disease type 1. In the European Union,…