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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5600096/
https://www.ncbi.nlm.nih.gov/pubmed/28569047
http://dx.doi.org/10.1002/ajh.24801

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5600096/
https://www.ncbi.nlm.nih.gov/pubmed/28569047
http://dx.doi.org/10.1002/ajh.24801

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