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

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...

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Detalhes bibliográficos
Publicado no:Blood
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Clinical Trials and Observations
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5409450/
https://ncbi.nlm.nih.gov/pubmed/28167660
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2016-12-758409
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