Gaucher disease: a systematic review and meta-analysis of bone complications and their response to treatment

@article{Piran2010GaucherDA,
  title={Gaucher disease: a systematic review and meta-analysis of bone complications and their response to treatment},
  author={Siavash Piran and Dominick J. Amato},
  journal={Journal of Inherited Metabolic Disease},
  year={2010},
  volume={33},
  pages={271-279}
}
Type 1 Gaucher disease (GD1) is an inherited lysosomal storage disease, which is often managed by enzyme replacement therapy (ERT). The bone response to ERT is usually slower than visceral and hematological responses. There is uncertainty as to whether an increase in the dosage of ERT has a beneficial effect. The aim of our study was to determine whether or not there is sufficient evidence to make a definitive statement about the effects of ERT and substrate reduction therapy (SRT) on bone… CONTINUE READING

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