Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.

Orphanet journal of rare diseases

Volumn 7, Issue , 2012, Pages

  Germain, Dominique P ^a   Giugliani, Roberto ^b   Hughes, Derralynn A ^b   Mehta, Atul ^b   Nicholls, Kathy ^b   Barisoni, Laura ^b   Jennette, Charles J ^b   Bragat, Alexander ^b   Castelli, Jeff ^b   Sitaraman, Sheela ^b   Lockhart, David J ^b   Boudes, Pol F ^b  

^a LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; CERAMIDE TRIHEXOSIDE; CHAPERONE; GLOBOTRIAOSYLCERAMIDE;

ARTICLE; ENZYMOLOGY; FABRY DISEASE; HUMAN; MALE; METABOLISM; PATHOLOGY; PHASE 2 CLINICAL TRIAL;

ALPHA-GALACTOSIDASE; FABRY DISEASE; HUMANS; MALE; MOLECULAR CHAPERONES; TRIHEXOSYLCERAMIDES;

MLCS; MLOWN;

EID: 84869875424     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-7-91     Document Type: Article

References (0)