Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.

@article{Case2008ImprovementWO,
  title={Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.},
  author={Laura Case and Dwight D. Koeberl and Sarah P. Young and Deeksha M. Bali and Stephanie M Dearmey and Joanne F Mackey and Priya Kishnani},
  journal={Molecular genetics and metabolism},
  year={2008},
  volume={95 4},
  pages={233-5}
}
Benefits of enzyme replacement therapy with Myozyme (alglucosidase alfa), anecdotally reported in late-onset Pompe disease, range from motor and pulmonary improvement in less severely affected patients, to stabilization with minimal improvement in those with advanced disease. We report a case of a 63-year-old patient with significant morbidity who made notable motor and pulmonary function gains after two years on therapy. Thus, improvements in those with advanced disease may be possible after… CONTINUE READING

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