Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.
Mistry, Pramod K; Balwani, Manisha; Baris, Hagit N; Turkia, Hadhami Ben; Burrow, T Andrew; Charrow, Joel; Cox, Gerald F; Danda, Sumita; Dragosky, Marta; Drelichman, Guillermo; El-Beshlawy, Amal; Fraga, Cristina; Freisens, Selena; Gaemers, Sebastiaan; Hadjiev, Evgueniy; Kishnani, Priya S; Lukina, Elena; Maison-Blanche, Pierre; Martins, Ana Maria; Pastores, Gregory; Petakov, Milan; Peterschmitt, M Judith; Rosenbaum, Hanna; Rosenbloom, Barry; Underhill, Lisa H; Cox, Timothy M.
Blood Cells Mol Dis
; 77: 101-102, 2019 07.
Artigo em Inglês | MEDLINE | ID: mdl-31029022
Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States.
Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.
Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial.
A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration.
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.