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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Detalles Bibliográficos
Autores principales: Wagner, Victoria F., Northrup, Hope, Hashmi, S. Shahrukh, Nguyen, Joanne M., Koenig, Mary Kay, Davis, Jessica M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2017
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5794801/
https://www.ncbi.nlm.nih.gov/pubmed/28803392
http://dx.doi.org/10.1007/s10897-017-0137-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5794801/
https://www.ncbi.nlm.nih.gov/pubmed/28803392
http://dx.doi.org/10.1007/s10897-017-0137-0

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