Innovative strategies to treat protein misfolding in inborn errors of metabolism: pharmacological chaperones and proteostasis regulators

Author:

Muntau Ania C.,Leandro João,Staudigl Michael,Mayer Felix,Gersting Søren W.

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Reference159 articles.

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2. Amicus Therapeutics. (2012). Amicus Therapeutics and GlaxoSmithKline announce top line 6-month primary treatment period results from first phase 3 Fabry monotherapy study. Retrieved November 3, from http://ir.amicustherapeutics.com/releasedetail.cfm?ReleaseID=727915

3. Amicus Therapeutics. (2013). Preclinical Programs. Retrieved November 3, from http://www.amicusrx.com/preclinical.aspx

4. Anfinsen CB (1973) Principles that govern the folding of protein chains. Science 181:223–230

5. Arakawa T, Ejima D, Kita Y, Tsumoto K (2006) Small molecule pharmacological chaperones: From thermodynamic stabilization to pharmaceutical drugs. Biochim Biophys Acta 1764:1677–1687

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