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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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Detalles Bibliográficos
Autores principales: Picache, Jaqueline A., Zheng, Wei, Chen, Catherine Z.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9294361/
https://www.ncbi.nlm.nih.gov/pubmed/35865957
http://dx.doi.org/10.3389/fphar.2022.906647