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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...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer US
2017
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| Materias: | |
| 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 |