The outcome of clinical parameters in adults with severe Type I Gaucher disease using very low dose enzyme replacement therapy.

@article{Wilson2007TheOO,
  title={The outcome of clinical parameters in adults with severe Type I Gaucher disease using very low dose enzyme replacement therapy.},
  author={Callum Wilson and Ruth Spearing and Lochie Teague and Patsy Robertson and Hilary A Blacklock},
  journal={Molecular genetics and metabolism},
  year={2007},
  volume={92 1-2},
  pages={131-6}
}
Enzyme replacement therapy is now well established as the treatment of choice in Type I Gaucher disease. Historically higher dosage regimens have been used in preference to lower doses despite the little clinical evidence in the way of large controlled clinical trials to support this. Moreover, the extraordinary cost of therapy means that not all eligible patients are able to be treated at the higher dose. Twelve type I adult patients with relatively severe disease were commenced on a very low… CONTINUE READING

From This Paper

Topics from this paper.
4 Citations
0 References
Similar Papers

Citations

Publications citing this paper.
Showing 1-4 of 4 extracted citations

Similar Papers

Loading similar papers…