Now showing items 1-2 of 2

    • Bilateral symmetrical cortical osteolytic lesions in two patients with Gaucher disease 

      Medina Canon, Astrid; Oppenheim, Iam M.; Barcenas, William; Groden, Catherine; Goker-Alpan, Ozlem; Resnik, Charles S.; Sidransky, Ellen (International Skeletal SocietyBerlín, Alemania, 2011-09-20)
      Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder characterized by the reduced or absent activity of glucocerebrosidase. The disease is split into three types. Type 3, or chronic neuronopathic ...
    • 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 ...