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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...
Tallennettuna:
| Julkaisussa: | Orphanet J Rare Dis |
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| Päätekijät: | , , , |
| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
BioMed Central
2015
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| Aiheet: | |
| Linkit: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4404669/ https://ncbi.nlm.nih.gov/pubmed/25896727 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0262-5 |
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