Gaucher disease: Progress and ongoing challenges.

Abstract excerpt

Over the past decades, tremendous progress has been made in the field of Gaucher disease, the inherited deficiency of the lysosomal enzyme glucocerebrosidase. Many of the colossal achievements took place during the course of the sixty-year tenure of Dr. Roscoe Brady at the National Institutes of Health. These include the recognition of the enzymatic defect involved, the isolation and characterization of the...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Enzyme Replacement Therapy
• Share to Gaucher Disease
• Share to Genetic Therapy