Therapeutic Strategies For Tay-Sachs Disease

Therapeutic Strategies For Tay-Sachs Disease

Tay-Sachs disease (TSD) is an autosomal recessive disease that features progressive neurodegenerative presentations. It affects one in 100,000 live births. Currently, there is no approved therapy or cure. This review summarizes multiple drug development strategies for TSD, including enzyme replaceme...

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Bibliographic Details
Main Authors: Jaqueline A. Picache, Wei Zheng, Catherine Z. Chen
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-07-01
Series:Frontiers in Pharmacology
Subjects:
drug development
enzymatic assay
phenotypic screen
high throughput screen (HTS)
mass spectrometry
tay-sachs disease (TSD)
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2022.906647/full

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https://www.frontiersin.org/articles/10.3389/fphar.2022.906647/full

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