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Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics
Lysosomal storage diseases (LSDs) are a group of genetic disorders due to defects in any aspect of lysosomal biology. During the past two decades, different approaches have been introduced for the treatment of these conditions. Among them, enzyme replacement therapy (ERT) represented a major advance...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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WILEY-VCH Verlag
2009
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3378140/ https://www.ncbi.nlm.nih.gov/pubmed/20049730 http://dx.doi.org/10.1002/emmm.200900036 |