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Treatment of profound thrombocytopenia in a patient with Gaucher disease type 1: Is there a role for substrate reduction therapy

The availability of three enzyme replacement therapy (ERT) drugs and two substrate reduction therapy (SRT) drugs to treat Gaucher disease provides an opportunity to tailor therapies to a patient's specific clinical concerns. However, there is a gap in the literature regarding individual drug ef...

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Detalles Bibliográficos
Autores principales:	Ha, Christine I., DeArmey, Stephanie, Cope, Heidi, Rairikar, Mugdha, Kishnani, Priya S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2017
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5489868/ https://www.ncbi.nlm.nih.gov/pubmed/28702360 http://dx.doi.org/10.1016/j.ymgmr.2017.06.003

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