Φορτώνει......
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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Orphanet J Rare Dis |
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| Κύριοι συγγραφείς: | , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
BioMed Central
2015
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| Θέματα: | |
| Διαθέσιμο Online: | 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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