Effect of Oral Eliglustat on Splenomegalyin Patients With Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial

Mistry el al determine whether eliglustat, a novel oral substrate reduction therapy, safely reverses clinical manifestations in untreated adults with Gaucher disease type 1. Among previously untreated adults with Gaucher disease type 1, treatment with eliglustat compared with placebo for 9 months re...

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Bibliographic Details
Published in:JAMA : the journal of the American Medical Association 2015-02, Vol.313 (7), p.695
Main Authors: 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, Balwani, Manisha, Danda, Sumita, Hadjiev, Evgueniy, Ortega, Andres, Shankar, Suma, Solano, Maria Helena, Ross, Leorah, Angell, Jennifer, Peterschmitt, M Judith
Format: Article
Language:English
Subjects:
Adults
Clinical trials
Disease
Therapy
Online Access:Get full text
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Summary:Mistry el al determine whether eliglustat, a novel oral substrate reduction therapy, safely reverses clinical manifestations in untreated adults with Gaucher disease type 1. Among previously untreated adults with Gaucher disease type 1, treatment with eliglustat compared with placebo for 9 months resulted in significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count.
ISSN:0098-7484
1538-3598