Enzyme replacement therapy and beyond—in memoriam Roscoe O. Brady, M.D. (1923–2016)

@article{Ries2017EnzymeRT,
  title={Enzyme replacement therapy and beyond—in memoriam Roscoe O. Brady, M.D. (1923–2016)},
  author={M. Ries},
  journal={Journal of Inherited Metabolic Disease},
  year={2017},
  volume={40},
  pages={343-356}
}
  • M. Ries
  • Published 2017
  • Medicine
  • Journal of Inherited Metabolic Disease
Lysosomal storage disorders are strong candidates for the development of specific innovative therapies. The discovery of enzyme deficiencies is an important milestone in understanding the underlying cause of disease. Being able to replace the first missing enzyme in a lysosomal storage required three decades of dedicated research. Successful drug development for lysosomal storage disorders was fostered by the U.S. Orphan Drug Act. Various optimization strategies have the potential to overcome… Expand
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