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Clinical studies in lysosomal storage diseases: Past, present, and future
Lysosomal storage disorders (LSDs) consist of over 40 diseases, some of which are amenable to treatment. In this review, we consider the regulatory context in which LSDs studies are performed, highlight design specificities and explore operational challenges. Orphan drug legislations, both in Europe...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Landes Bioscience
2013
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3915565/ https://www.ncbi.nlm.nih.gov/pubmed/25003011 http://dx.doi.org/10.4161/rdis.26690 |