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Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1. The ENGAGE Randomized Clinical Trial

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 unt...

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Bibliographic Details
Institution:FUCS -Fundación Universitaria de Ciencias de la Salud
Main Authors: 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
Format: Artículo de revista
Language:English
Published: American Medical Association 2015-02-17
Subjects:
Eliglustat
Esplenomegalia
Trombocitopenia
Enfermedad esquelética
Enfermedad de gaucher
Anemia
Inhibidores enzimáticos
Online Access:https://repositorio.fucsalud.edu.co/handle/001/1868
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