Gaucher Disease, Type 2 research hub

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

This study is testing a new treatment, LY3884961, for babies with a serious inherited condition called Type 2 Gaucher disease. Researchers want to see if it's safe and effective. It's a long-term study, following babies for up to 5 years.