Article
Ten plus one challenges in diseases of the lysosomal system.
Molecular genetics and metabolism - 1 Jan 2000
Grabowski Gregory A, Whitley Chester
Abstract excerpt
The advent of the first effective specific therapy for a lysosomal storage disease (LSDs), Gaucher disease type 1, by Roscoe O. Brady was foundational for development of additional treatments for this group of rare diseases. The past 26years, since the approval of enzyme therapy for Gaucher disease type 1, have witnessed a burgeoning understanding of LSDs at genetic, molecular, biochemical, cell biologic, and...
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