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Datamonitor Healthcare CV&Met: Lysosomal Storage Disorders Market Spotlight

May 03, 2021

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.

Indications Covered: Alpha-mannosidosis
Cystinosis
Fabry's Disease
Farber Disease
Gaucher's Disease
Glycogen Storage Disease (GSD)
GM1 Gangliosidosis
GM2 Gangliosidoses (Tay-Sachs Disease, Sandhoff Disease, AB Variant)
Krabbe Disease (Globoid Cell Leukodystrophy)
Lysosomal Acid Lipase Deficiency
Metachromatic Leukodystrophy
Mucopolysaccharidosis I (MPS I; Hurler Syndrome)
Mucopolysaccharidosis II (MPS II; Hunter Syndrome)
Mucopolysaccharidosis III - Subtype Undisclosed (MPS III; Sanfilippo Syndrome)
Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A Syndrome)
Mucopolysaccharidosis IIIB (MPS IIIB; Sanfilippo B Syndrome)
Mucopolysaccharidosis IV (MPS IV; Morquio Syndrome)
Mucopolysaccharidosis VI (MPS VI)
Mucopolysaccharidosis VII (MPS VII; Sly Syndrome)
Neuronal Ceroid Lipofuscinosis (NCL)
Niemann-Pick Disease
Pompe Disease
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