Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act

@inproceedings{Mechler2015PressureFD,
  title={Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act},
  author={Konstantin Mechler and William K. Mountford and Georg Friedrich Hoffmann and Markus Ries},
  booktitle={Orphanet journal of rare diseases},
  year={2015}
}
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 premature death. More than 30 years ago the Orphan Drug Act of 1983 passed the United States legislation intended to facilitate the development of drugs for rare disorders. We directed our efforts in… CONTINUE READING
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