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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Bibliographic Details
Published in:Mol Genet Metab Rep
Main Authors: Ha, Christine I., DeArmey, Stephanie, Cope, Heidi, Rairikar, Mugdha, Kishnani, Priya S.
Format: Artigo
Language:Inglês
Published: Elsevier 2017
Subjects:
Case Report
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5489868/
https://ncbi.nlm.nih.gov/pubmed/28702360
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.06.003
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