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Progress in the Development of Small Molecule Therapeutics for the Treatment of Neuronal Ceroid Lipofuscinoses (NCLs).

Abstract：

:The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited and incurable neurodegenerative disorders primarily afflicting the pediatric population. Current treatment regimens offer only symptomatic relief and do not target the underlying cause of the disease. Although the underlying pathophysiology that drives disease progression is unknown, several small molecules have been identified with diverse mechanisms of action that provide promise for the treatment of this devastating disease. This review aims to summarize the current cellular and animal models available for the identification of potential therapeutics and presents the current state of knowledge on small molecule compounds that demonstrate in vitro and/or in vivo efficacy across the NCLs with an emphasis on targets of action.

journal_name

J Med Chem

journal_title

Journal of medicinal chemistry

authors

Kinarivala N,Trippier PC

doi

10.1021/acs.jmedchem.5b01020

subject

Has Abstract

pub_date

2016-05-26 00:00:00

pages

4415-27

issue

10

eissn

0022-2623

issn

1520-4804

journal_volume

59

pub_type

杂志文章,评审

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