Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.

K Mistry, Pramod; Lukina, Elena; Ben Turkia, Hadhami; Amato, Dominick; Baris, Hagit; Dasouki, Majed; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Pastores, Gregory; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Shankar, Suma; Helena Solano, Maria; Ross, Leorah; Angell, Jennifer; Judith Peterschmitt, M

doi:10.1001/jama.2015.459

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Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial.

Shared by NobleBlocks on Feb 17, 2015 • 12:00 AM UTC

Authors:

Pramod K Mistry

Elena Lukina

Hadhami Ben Turkia

Abstract

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 oral substrate reduction therapy, safely reverses clinical manifestations in untrea...

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