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Optimal therapy in Gaucher disease
Gaucher disease (GD), the inherited deficiency of the lysosomal enzyme glucocerebrosidase, presents with a wide range of symptoms of varying severity, and primarily affects the skeletal, hematologic and nervous systems. To date, the standard of care has included enzyme replacement therapy with imigl...
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| Formato: | Texto |
| Lenguaje: | English |
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Dove Medical Press
2010
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2909498/ https://www.ncbi.nlm.nih.gov/pubmed/20668714 |