Safety and pharmacodynamic effects of a pharmacological chaperone on &#945;-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.

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

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.

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

Afiliación

Germain DP; Division of Medical Genetics, Hôpital Raymond Poincaré (AP-HP), University of Versailles - St Quentin en Yvelines (UVSQ), Garches, 92380, France. dominique.germain@rpc.aphp.fr

Orphanet J Rare Dis ; 7: 91, 2012 Nov 24.

Article en En | MEDLINE | ID: mdl-23176611

Asunto(s)

Enfermedad de Fabry/tratamiento farmacológico; Chaperonas Moleculares/uso terapéutico; Trihexosilceramidas/metabolismo; alfa-Galactosidasa/metabolismo; Enfermedad de Fabry/enzimología; Enfermedad de Fabry/metabolismo; Enfermedad de Fabry/patología; Humanos; Masculino; Chaperonas Moleculares/efectos adversos

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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Trihexosilceramidas / Enfermedad de Fabry / Alfa-Galactosidasa / Chaperonas Moleculares Límite: Humans / Male Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2012 Tipo del documento: Article País de afiliación: Francia

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