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脆性 X 综合征:治疗方法的研究进展。

Fragile X syndrome: an update on developing treatment modalities.

机构信息

Seaside Therapeutics, 840 Memorial Drive, Cambridge, Masssachusetts 02139, USA.

出版信息

ACS Chem Neurosci. 2011 Aug 17;2(8):402-10. doi: 10.1021/cn200019z. Epub 2011 Mar 22.

Abstract

Intellectual disability (ID; mental retardation) is considered an immutable condition. Current medical practices are aimed at relieving symptoms and not at altering the underlying cognitive deficits. Scientific advancements from the past decade have led to the exciting possibility that ID may now be treatable. Moreover, pharmaceutical therapies targeting the most common form of inherited ID, Fragile X syndrome (FXS), may become the new benchmark for central nervous system (CNS) drug discovery: seeking cures for neurodevelopmental disorders.

摘要

智力残疾(ID;智力迟钝)被认为是一种不可改变的状况。目前的医疗实践旨在缓解症状,而不是改变潜在的认知缺陷。过去十年的科学进步带来了一个令人兴奋的可能性,即 ID 现在可能是可治疗的。此外,针对最常见的遗传性 ID 形式——脆性 X 综合征(FXS)的药物疗法可能成为中枢神经系统(CNS)药物发现的新基准:寻找神经发育障碍的治疗方法。

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