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...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Orphanet J Rare Dis
Hauptverfasser: Mechler, Konstantin, Mountford, William K, Hoffmann, Georg F, Ries, Markus
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2015
Schlagworte:
Research
Online Zugang: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
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!

Ähnliche Einträge