Free Access
| Issue |
Med Sci (Paris)
Volume 29, Number 6-7, Juin–Juillet 2013
|
|
|---|---|---|
| Page(s) | 579 - 582 | |
| Section | Nouvelles | |
| DOI | https://doi.org/10.1051/medsci/2013296009 | |
| Published online | 12 July 2013 | |
- Germain DP. Fabry disease. Orphanet J Rare Dis 2010 ; 5 : 30. [CrossRef] [PubMed] [Google Scholar]
- Eng CM, Guffon N, Wilcox WR, et al. Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry’s disease. N Eng J Med 2001 ; 345 : 9–16. [CrossRef] [Google Scholar]
- Schiffmann R, Kopp JB, Austin HA, 3rd, et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001 ; 285 : 2743–2749. [CrossRef] [PubMed] [Google Scholar]
- Mendre C, Mouillac B. Chaperons pharmacologiques. Med Sci (Paris) 2010 ; 26 : 627–635. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
- Fan JQ, Ishii S, Asano N, Suzuki Y. Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor. Nat Med 1999 ; 5 : 112–115. [CrossRef] [PubMed] [Google Scholar]
- Germain DP, Fan JQ. Pharmacological chaperone therapy by active-site-specific chaperones in Fabry disease: in vitro and preclinical studies. Int J Clin Pharmacol and Ther 2009 ; 47 : S111–S117. [Google Scholar]
- Germain DP, Giugliani R, Hughes DA, et al. Safety, pharmacodynamic effects of a pharmacological chaperone on alpha-galactosidase A activity, globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis 2012 ; 7 : 91. [CrossRef] [PubMed] [Google Scholar]
- Wu X, Katz E, Della Valle MC, et al. A pharmacogenetic approach to identify mutant forms of alpha-galactosidase A that respond to a pharmacological chaperone for Fabry disease. Hum Mut 2011 ; 32 : 965–977. [CrossRef] [Google Scholar]
- Guce AI, Clark NE, Rogich JJ, Garman SC. The molecular basis of pharmacological chaperoning in human alpha-galactosidase. Chem Biol 2011 ; 18 : 1521–1526. [CrossRef] [PubMed] [Google Scholar]
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