Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry

This study tests the hypothesis that the prevalence of severe clinical manifestations in Gaucher disease type 1 (GD1) patients at the time of treatment initiation has changed since alglucerase/imiglucerase enzyme replacement therapy (ERT) was approved in the United States (US) in 1991. US algluceras...

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Bibliografski detalji
Izdano u:Am J Hematol
Glavni autori: Mistry, Pramod K., Batista, Julie L., Andersson, Hans C., Balwani, Manisha, Burrow, Thomas Andrew, Charrow, Joel, Kaplan, Paige, Khan, Aneal, Kishnani, Priya S., Kolodny, Edwin H., Rosenbloom, Barry, Scott, C. Ronald, Weinreb, Neal
Format: Artigo
Jezik:Inglês
Izdano: John Wiley and Sons Inc. 2017
Teme:
Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5600096/
https://ncbi.nlm.nih.gov/pubmed/28569047
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.24801
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