Tag Archives: lysosomal storage disease treatment

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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