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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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Podrobná bibliografie
Vydáno v:Mol Genet Metab Rep
Hlavní autoři: Ha, Christine I., DeArmey, Stephanie, Cope, Heidi, Rairikar, Mugdha, Kishnani, Priya S.
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2017
Témata:
On-line přístup: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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