Tag Archives: Lysosomal Storage Disorder Therapeutics

Lysosomal Storage Disorder Therapeutics Pipeline Analysis 2017

Lysosomal storage disorder refers to inherited metabolic disorders that are characterize by enzyme deficiencies, which results in an abnormal build-up of various toxic materials in the body’s cells. There are nearly 50 types of lysosomal storage disorder which affect various parts of the body including the brain, skeleton, heart, central nervous system and skin.

Some of the lysosomal storage disorders are Aspartylglucosaminuria, Batten Disease, Cystinosis, Fabry Disease, Glycogen Storage Disease II (Pompe Disease), GM2-Gangliosidosis Type I (Tay Sachs Disease), GM2-Gangliosidosis Type II (Sandhoff Disease), Metachromatic Leukodystrophy, Gaucher’s Disease Types I, II, and III, Mucolipidosis Types I, II/III and IV, Mucopolysaccharide Storage Diseases (Hurler Disease and variants, Hunter, Sanfilippo Types A,B,C,D, Morquio Types A and B, Maroteaux-Lamy and Sly diseases), Niemann-Pick Disease Types A/B, C1 and C2 and Schindler Disease Types I and II.

Lysosomal Storage Disorder Therapeutics – Clinical Trials & Results, 2017

The lysosomal storage disorder therapeutics pipeline comprises approximately 74 drug candidates in different stages of development.

According to the research findings, most of the drug candidates for lysosomal storage disorder pipeline are being developed to be administered by intravenous route.

Browse Report Description at: https://www.psmarketresearch.com/market-analysis/lysosomal-storage-disorders-pipeline-analysis

Amicus Therapeutics, Inc. is using chaperone advanced replacement therapy (CHART) technology platform for the development of their drug candidates for the treatment of Fabry disease, a type of lysosomal storage disorder. In a chaperone-advanced replacement therapy, a unique pharmacological chaperone, binds to the infused therapeutic enzyme, stabilizing the enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake, greater lysosomal activity, more reduction of storage material, and lower immunogenicity compared to ERT alone.

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