Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

@article{Sun2007EnhancedRT,
  title={Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.},
  author={Baodong Sun and Andrew Bird and Sarah P. Young and Priya Kishnani and Y-T Chen and Dwight D. Koeberl},
  journal={American journal of human genetics},
  year={2007},
  volume={81 5},
  pages={1042-9}
}
Pompe disease, which results from mutations in the gene encoding the glycogen-degrading lysosomal enzyme acid alpha -glucosidase (GAA) (also called "acid maltase"), causes death in early childhood related to glycogen accumulation in striated muscle and an accompanying infantile-onset cardiomyopathy. The efficacy of enzyme replacement therapy (ERT) with recombinant human GAA was demonstrated during clinical trials that prolonged subjects' overall survival, prolonged ventilator-free survival, and… CONTINUE READING