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 untreate...

Full description

Autores:

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

Tipo de recurso:

Article of journal

Fecha de publicación:

2015

Institución:

Fundación Universitaria de Ciencias de la Salud - FUCS

Repositorio:

Repositorio Digital Institucional ReDi

Idioma:

eng

OAI Identifier:

oai:repositorio.fucsalud.edu.co:001/1868

Acceso en línea:

https://repositorio.fucsalud.edu.co/handle/001/1868

Palabra clave:

Eliglustat
Esplenomegalia
Trombocitopenia
Enfermedad esquelética
Enfermedad de gaucher
Anemia
Inhibidores enzimáticos

Rights

openAccess

License

Atribución-NoComercial-SinDerivadas 4.0 Internacional (CC BY-NC-ND 4.0)