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...

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Detalles Bibliográficos
Autores principales: Mechler, Konstantin, Mountford, William K, Hoffmann, Georg F, Ries, Markus
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Research
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

Internet

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

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