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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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Dettagli Bibliografici
Pubblicato in:J Genet Couns
Autori principali: Wagner, Victoria F., Northrup, Hope, Hashmi, S. Shahrukh, Nguyen, Joanne M., Koenig, Mary Kay, Davis, Jessica M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer US 2017
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Accesso online: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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