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TITLE: Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease
AUTHORS: Biegstraaten, M; Cox, TM; Belmatoug, N; Berger, MG; Collin Histed, T; Vom Dahl, SV; Di Rocco, M; Fraga, C; Giona, F; Giraldo, P; Hasanhodzic, M; Hughes, DA; Iversen, PO; Kiewiet, AI; Lukina, E; Machaczka, M; Marinakis, T; Mengel, E; Pastores, GM; Ploeckinger, U; Rosenbaum, H; Serratrice, C; Symeonidis, A; Szer, J; Timmerman, J; Tylki Szymanska, A; Weisz W Hubshman; Zafeiriou, DI; a Zimran; Hollak, CEM; ...More
PUBLISHED: 2018, SOURCE: BLOOD CELLS MOLECULES AND DISEASES, VOLUME: 68
INDEXED IN: WOS
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TITLE: Chronic kidney disease and an uncertain diagnosis of Fabry disease: Approach to a correct diagnosis  Full Text
AUTHORS: Linda van der Tol; Einar Svarstad; Alberto Ortiz; Camilla Tondel; Oliveira, JP ; Liffert Vogt; Stephen Waldek; Derralynn A Hughes; Robin H Lachmann; Wim Terryn; Carla E Hollak; Sandrine Florquin; Marius van den Bergh Weerman; Christoph Wanner; Michael L West; Marieke Biegstraaten; Gabor E Linthorst;
PUBLISHED: 2015, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 114, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef: 46
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TITLE: Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document  Full Text
AUTHORS: Marieke Biegstraaten; Reynir Arngrimsson; Frederic Barbey; Lut Boks; Franco Cecchi; Patrick B Deegan; Ulla Feldt Rasmussen; Tarekegn Geberhiwot; Dominique P Germain; Chris Hendriksz; Derralynn A Hughes; Ilkka Kantola; Nesrin Karabul; Christine Lavery; Gabor E Linthorst; Atul Mehta; Erica van de Mheen; Oliveira, JP ; Rossella Parini; Uma Ramaswami; Michael Rudnicki; Andreas Serra; Claudia Sommer; Gere Sunder Plassmann; Einar Svarstad; Annelies Sweeb; Wim Terryn; Anna Tylki Szymanska; Camilla Tondel; Bojan Vujkovac; Frank Weidemann; Frits A Wijburg; Peter Woolfson; Carla E M Hollak; ...More
PUBLISHED: 2015, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 10, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef: 234
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TITLE: Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease  Full Text
AUTHORS: Carla E M Hollak; Stephan vom Dahl; Johannes M F G Aerts; Nadia Belmatoug; Bruno Bembi; Yossi Cohen; Tanya Collin Histed; Patrick Deegan; Laura van Dussen; Pilar Giraldo; Eugen Mengel; Helen Michelakakis; Jeremy Manuel; Martin Hrebicek; Rosella Parini; Joerg Reinke; Maja di Rocco; Miguel Pocovi; Maria Clara S Sa Miranda ; Anna Tylki Szymanska; Ari Zimran; Timothy M Cox; ...More
PUBLISHED: 2010, SOURCE: BLOOD CELLS MOLECULES AND DISEASES, VOLUME: 44, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef