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Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials.
Elstein, Deborah; Mellgard, Björn; Dinh, Quinn; Lan, Lan; Qiu, Yongchang; Cozma, Claudia; Eichler, Sabrina; Böttcher, Tobias; Zimran, Ari.
Afiliação
  • Elstein D; Shire, Zählerweg 10, 6300 Zug, Switzerland. Electronic address: delstein-c@shire.com.
  • Mellgard B; Shire, 300 Shire Way, Lexington, MA, USA. Electronic address: bjorn.mellgard@Shire.com.
  • Dinh Q; Shire, 300 Shire Way, Lexington, MA, USA. Electronic address: quinndinh1@yahoo.com.
  • Lan L; Shire, 300 Shire Way, Lexington, MA, USA. Electronic address: Lan.lan@Shire.com.
  • Qiu Y; Shire, 300 Shire Way, Lexington, MA, USA. Electronic address: yqiu@Shire.com.
  • Cozma C; Centogene AG, Schillingallee 68, 18057 Rostock, Germany. Electronic address: Claudia.Cozma@centogene.com.
  • Eichler S; Centogene AG, Schillingallee 68, 18057 Rostock, Germany. Electronic address: Sabrina.Eichler@centogene.com.
  • Böttcher T; Centogene AG, Schillingallee 68, 18057 Rostock, Germany. Electronic address: Tobias.Boettcher@centogene.com.
  • Zimran A; Gaucher Clinic, Shaare Zedek Medical Center, the Hebrew University-Hadassah Medical School, Shmu'el Bait St 12, Jerusalem, Israel. Electronic address: azimran@gmail.com.
Mol Genet Metab ; 122(1-2): 113-120, 2017 09.
Article em En | MEDLINE | ID: mdl-28851512

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Esfingolipídeos / Glicolipídeos / Biomarcadores / Doença de Gaucher / Glucosilceramidase Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Esfingolipídeos / Glicolipídeos / Biomarcadores / Doença de Gaucher / Glucosilceramidase Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2017 Tipo de documento: Article