Now showing items 1-2 of 2

    • Consensus Conference: A reappraisal of Gaucher disease - diagnosis and disease management algorithms 

      Cappellini, María Domenica; Lukina, Elena; Özsan, Hayri; Mistry, Pramod K.; Mach Pascual, Sara; Rosenbaum, Hanna; Solano, María Helena; Spigelman, Zachary; Villarrubia, Jesús; Watman, Nora Patricia; Massenkeil, Gero (2010-09-23)
    • Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1. The ENGAGE Randomized Clinical Trial 

      Solano Trujillo, María Helena; Mistry, Pramod K; Lukina, Elena; Turkia, Hadhami Ben; Amato, Dominick; Baris, Hagit; Dasouki, Majed; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Pastores, Gregory; Petakov, Milan; Assouline, Sarit; Balwan, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Shanka, Suma; Ross, Leorah; Angel, Jennifer; Peterschmitt, Judith (American Medical AssociationEstados Unidos, 2015-02-17)
      Importance Gaucher disease type 1 is characterized by hepatosplenomegaly, anemia, thrombocytopenia, and skeletal disease. A safe, effective oral therapy is needed. Objective To determine whether eliglustat, a novel ...