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TITLE: Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1  Full Text
AUTHORS: Pramod K Mistry; Manisha Balwani; Hagit N Baris; Hadhami Ben Turkia; Andrew A Burrow; Joel Charrow; Gerald F Cox; Sumita Danda; Marta Dragosky; Guillermo Drelichman; Amal El Beshlawy; Cristina Fraga; Selena Freisens; Sebastiaan Gaemers; Evgueniy Hadjiev; Priya S Kishnani; Elena Lukina; Pierre Maison Blanche; Ana Maria Martins; Gregory Pastores; Milan Petakov; Judith J Peterschmitt; Hanna Rosenbaum; Barry Rosenbloom; Lisa H Underhill; Timothy M Cox; ...More
PUBLISHED: 2019, SOURCE: BLOOD CELLS MOLECULES AND DISEASES, VOLUME: 77
INDEXED IN: Scopus WOS
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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: 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