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Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act
BACKGROUND: Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of high morbidity often associated with prematur...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4404669/ https://www.ncbi.nlm.nih.gov/pubmed/25896727 http://dx.doi.org/10.1186/s13023-015-0262-5 |