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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Am J Hematol
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: John Wiley and Sons Inc. 2017
Θέματα:
Research Articles
Διαθέσιμο Online: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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