An innovative approach to the treatment of Gaucher disease and possibly other metabolic disorders of the brain.

Brady, Roscoe O

An innovative approach to the treatment of Gaucher disease and possibly other metabolic disorders of the brain. [electronic resource] - Journal of inherited metabolic disease May 2013 - 451-4 p. digital

Publication Type: Journal Article; Review

1573-2665

10.1007/s10545-012-9515-9 doi


Blood-Brain Barrier--metabolism
Brain Diseases, Metabolic--genetics
Enzyme Replacement Therapy--methods
Gaucher Disease--genetics
Glucosylceramidase--deficiency
Humans
Models, Biological
Mutation--physiology
Therapies, Investigational--methods