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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Berlin Heidelberg 2014
Materias:	Scientific Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4141971/ https://www.ncbi.nlm.nih.gov/pubmed/24816856 http://dx.doi.org/10.1007/s00256-014-1891-9

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