Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study

Blood Cells, Molecules, and Diseases - Tập 51 - Trang 116-124 - 2013
David J. Kuter1, Atul Mehta2, Carla E.M. Hollak3, Pilar Giraldo4, Derralynn Hughes5, Nadia Belmatoug6, Monika Brand7, Audrey Muller7, Berthold Schaaf8, Ruben Giorgino7, Ari Zimran9
1Massachusetts General Hospital, Boston, USA
2Royal Free Hospital, London, UK
3Academic Medical Centre, Amsterdam, The Netherlands
4Hospital Universitario Miguel Servet, Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Zaragoza, Spain
5Royal Free Hospital & University College Medical School, London, UK
6Hôpital Beaujon, Clichy, France
7Actelion Pharmaceuticals Ltd, Allschwil, Switzerland
8Factum GmbH, Offenbach, Germany
9Shaare Zedek Medical Center, Jerusalem, Israel

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Blood Cells, Molecules, and Diseases

Tập 51

116-124

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