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Showing results 1 to 5 of 5
Issue DateTitleAuthor(s)
1-Feb-2015ENCORE, a randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in Gaucher disease type 1 patients stabilized on enzyme replacement therapy: 24-month resultsCox, Timothy M.; Drelichman, Guillermo I.; Cravo, Renata; Balwani, Manisha; Burrow, Thomas Andrew; Martins, Ana Maria [UNIFESP]; Lukinag, Elena; Rosenbloom, Barry E.; Ross, Leorah H.; Angell, Jennifer; Peterschmitt, M. Judith; Addenbrookes Hosp; Hosp Ninos Dr Ricardo Gutierrez; HEMORIO; Mt Sinai Hosp; Cincinnati Childrens Hosp Med Ctr; Universidade Federal de São Paulo (UNIFESP); Minist Publ Hlth Russia; Cedars Sinai Tower Hematol Oncol Med Grp; Genzyme
1-Feb-2014ENCORE: A randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in Gaucher disease type 1 patients on enzyme replacement therapy who have reached therapeutic goalsCox, Timothy M.; Drelichman, Guillermo; Cravo, Renata; Balwani, Manisha; Burrow, Thomas Andrew; Martins, Ana Maria [UNIFESP]; Lukina, Elena; Rosenbloom, Barry; Ross, Leorah; Angell, Jennifer; Puga, Ana Cristina; Univ Cambridge; Hosp Ninos Dr Ricardo Gutierrez; HEMORIO; Mt Sinai Hosp; Cincinnati Childrens Hosp Med Ctr; Universidade Federal de São Paulo (UNIFESP); Minist Publ Hlth Russia; Cedars Sinai Tower Hematol Oncol; Genzyme
1-Feb-2014Female Fabry disease: significant improvement of Fabry disease-related gastrointestinal symptoms in a large cohort of female patients treated with agalsidase beta: data from the Fabry RegistryWilcox, William; Feldt-Rasmussen, Ulla; Martins, Ana Maria [UNIFESP]; Ortiz, Alberto; Weidemann, Frank; Lemay, Roberta; Hopkin, Robert; Cedars Sinai Med Ctr; Copenhagen Univ Hosp; Universidade Federal de São Paulo (UNIFESP); Univ Autonoma Madrid; Univ Klin Wurzburg; Genzyme; Cincinnati Childrens Hosp Med Ctr
1-Aug-2012Repression of osteocyte Wnt/beta-catenin signaling is an early event in the progression of renal osteodystrophySabbagh, Yves; Graciolli, Fabiana Giorgeti; O'Brien, Stephen; Tang, Wen; Reis, Luciene Machado dos; Ryan, Susan; Phillips, Lucy; Boulanger, Joseph; Song, Wenping; Bracken, Christina; Liu, Shiguang; Ledbetter, Steven; Dechow, Paul; Canziani, Maria Eugênia Fernandes [UNIFESP]; Carvalho, Aluizio Barbosa de [UNIFESP]; Jorgetti, Vanda; Moyses, Rosa M. A.; Schiavi, Susan C.; Genzyme; Universidade de São Paulo (USP); Baylor Coll Dent; Universidade Federal de São Paulo (UNIFESP)
1-Feb-2015Risk factors for severe clinical events and the incidence of these events in male and female patients with Fabry disease treated with agalsidase betaHopkin, Robert J.; Cabrera, Gustavo; Charrow, Joel; Lemay, Roberta; Martins, Ana Maria [UNIFESP]; Mauer, Michael; Ortiz, Alberto; Patel, Manesh R.; Sims, Katherine; Waldek, Stephen; Warnock, David G.; Wilcox, William R.; Cincinnati Childrens Hosp Med Ctr; Grp Med Viso; Ann & Robert H Lurie Childrens Hosp Chicago; Genzyme; Universidade Federal de São Paulo (UNIFESP); Univ Minnesota; IIS Fdn Jimenez Diaz UAM IRSIN REDINREN; Duke Univ; Harvard Univ; Univ Alabama Birmingham; Emory Univ