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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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Dades bibliogràfiques
Publicat a:J Genet Couns
Autors principals: Wagner, Victoria F., Northrup, Hope, Hashmi, S. Shahrukh, Nguyen, Joanne M., Koenig, Mary Kay, Davis, Jessica M.
Format: Artigo
Idioma:Inglês
Publicat: Springer US 2017
Matèries:
Accés en línia: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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