Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat

OBJECTIVE: Eliglustat is an investigational oral substrate reduction therapy for Gaucher disease type 1 (GD1). Its skeletal effects were evaluated by prospective monitoring of bone mineral density (BMD), fractures, marrow infiltration by Gaucher cells, focal bone lesions, and infarcts during an open...

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Detalhes bibliográficos
Main Authors: Kamath, Ravi S., Lukina, Elena, Watman, Nora, Dragosky, Marta, Pastores, Gregory M., Arreguin, Elsa Avila, Rosenbaum, Hanna, Zimran, Ari, Aguzzi, Rasha, Puga, Ana Cristina, Norfleet, Andrea M., Peterschmitt, M. Judith, Rosenthal, Daniel I.
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Berlin Heidelberg 2014
Assuntos:
Scientific Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4141971/
https://ncbi.nlm.nih.gov/pubmed/24816856
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00256-014-1891-9
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