Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

M. Cox, Timothy; Drelichman, Guillermo; Cravo, Renata; Balwani, Manisha; Andrew Burrow, Thomas; María Martins, Ana; Lukina, Elena; E. Rosenbloom, Barry; Göker-Alpan, Özlem; Watman, Nora; El‐Beshlawy, Amal; S. Kishnani, Priya; Lúcia Alves Pedroso, Maria; J.M. Gaemers, Sebastiaan; Tayag, Regina; Peterschmitt, Michel

doi:10.1182/blood-2016-12-758409

Public

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Published in Blood • Feb 7, 2017

Authors:

Timothy M. Cox

Guillermo Drelichman

Renata Cravo

Abstract

In the phase 3 Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE), at 1 year, eliglustat was noninferior to imiglucerase enzyme therapy in maintaining stable platelet counts, hemoglobin concentration...

Subject

Medicine

Enzyme replacement therapy

Adverse effect

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!