Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial

Gaucher disease type 1 is an autosomal recessive disorder caused by deficient activity of the lysosomal enzyme acid β-glucosidase resulting in accumulation of glucosylceramide and clinical manifestations of anemia, thrombocytopenia, hepatosplenomegaly, and skeletal disease. The historic standard of...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Pleat, Rebecca, Cox, Timothy M., Burrow, T. Andrew, Giraldo, Pilar, Goker-Alpan, Ozlem, Rosenbloom, Barry E., Croal, Laura R., Underhill, Lisa H., Gaemers, Sebastiaan J.M., Peterschmitt, M. Judith
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2016
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5050260/
https://ncbi.nlm.nih.gov/pubmed/27722092
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.08.009
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