Lysosomal storage disorders (LSDs) comprise a group of more than 70 metabolic diseases that are mainly inherited in an autosomal recessive manner. While the pathogenesis of the diseases is complex and not fully understood, each LSD is caused by mutations in genes encoding proteins that are necessary for lysosomal function, including lysosomal enzymes and lysosomal membrane proteins. Lysosomes are essential for recycling, signaling, and catabolism of macromolecules by digestive enzymes called hydrolases. Defects in these processes can lead to accumulation of undegraded or partially degraded macromolecules within the lysosomes, ultimately leading to cell dysfunction and cell death. 

This Datamonitor Healthcare report contains a Market Spotlight module.