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Drug repositioning can accelerate discovery of pharmacological chaperones.

ABSTRACT: A promising strategy for the treatment of genetic diseases, pharmacological chaperone therapy, has been proposed recently. It exploits small molecules which can be administered orally, reach difficult tissues such as the brain and have low cost. This strategy has a vast field of application. In order to make drug development as fast as possible, it is important to exploit drug repositioning. We evaluated the impact and limitations of this approach for rare diseases and we provide a shortcut in finding drugs for off-target usage.

SUBMITTER: Hay Mele B

PROVIDER: S-EPMC4429356 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature

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Drug repositioning can accelerate discovery of pharmacological chaperones.

Hay Mele Bruno B Citro Valentina V Andreotti Giuseppina G Cubellis Maria Vittoria MV

Orphanet journal of rare diseases 20150507

A promising strategy for the treatment of genetic diseases, pharmacological chaperone therapy, has been proposed recently. It exploits small molecules which can be administered orally, reach difficult tissues such as the brain and have low cost. This strategy has a vast field of application. In order to make drug development as fast as possible, it is important to exploit drug repositioning. We evaluated the impact and limitations of this approach for rare diseases and we provide a shortcut in f ...[more]

PMID: 25947946

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