Emptying the stores: lysosomal diseases and therapeutic strategies

Nature Reviews Drug Discovery 17, 133 (2018). doi:10.1038/nrd.2017.214 Author: Frances M. Platt Lysosomal storage disorders (LSDs) — designated as 'orphan' diseases — are inborn errors of metabolism caused by defects in genes that encode proteins involved in various aspects of lysosomal homeostasis. For many years, LSDs were viewed as unattractive targets for the development of therapies owing
Source: Nature Reviews Drug Discovery - Category: Drugs & Pharmacology Authors: Tags: Review Source Type: research