Enzyme replacement for lysosomal diseases.

@article{Brady2006EnzymeRF,
  title={Enzyme replacement for lysosomal diseases.},
  author={Roscoe O. Brady},
  journal={Annual review of medicine},
  year={2006},
  volume={57},
  pages={283-96}
}
Following the demonstration of the nature of the enzymatic defects in the sphingolipid storage disorders in the mid-1960s, consideration was directed to the development of therapy for patients with these conditions. High on the list of possibilities was enzyme supplementation or replacement. Many years of arduous investigation and the development of novel protein targeting strategies were required to bring this concept to fruition. Enzyme replacement therapy (ERT) was eventually shown to be… CONTINUE READING

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