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

Cox, Timothy M; Drelichman, Guillermo; Cravo, Renata; Balwani, Manisha; Burrow, Thomas Andrew; Martins, Ana Maria; Lukina, Elena; Rosenbloom, Barry; Goker-Alpan, Ozlem; Watman, Nora; El-Beshlawy, Amal; Kishnani, Priya S; Pedroso, Maria Lucia; Gaemers, Sebastiaan J M; Tayag, Regina; Peterschmitt, M Judith.
Blood; 129(17): 2375-2383, 2017 04 27.
Artigo em Inglês | MEDLINE | ID: mdl-28167660