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Attitudes of Individuals with Gaucher Disease toward Substrate Reduction Therapies

Type 1 Gaucher disease (GD) is the most common lysosomal storage disorder. Previously, treatment for GD was limited to intravenous enzyme replacement therapies (ERTs). More recently, oral substrate reduction therapies (SRTs) were approved for treatment of GD. Although both therapies alleviate diseas...

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Podrobná bibliografie
Vydáno v:J Genet Couns
Hlavní autoři: Wagner, Victoria F., Northrup, Hope, Hashmi, S. Shahrukh, Nguyen, Joanne M., Koenig, Mary Kay, Davis, Jessica M.
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer US 2017
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5794801/
https://ncbi.nlm.nih.gov/pubmed/28803392
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10897-017-0137-0
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