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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Bibliografiske detaljer
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.
Format: Artigo
Sprog:Inglês
Udgivet: Springer Berlin Heidelberg 2014
Fag:
Scientific Article
Online adgang: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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