Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail.

@article{Kikuchi1998ClinicalAM,
  title={Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail.},
  author={T. Kikuchi and H. W. Yang and M. Pennybacker and N. Ichihara and M. Mizutani and J. V. Van Hove and Y. Chen},
  journal={The Journal of clinical investigation},
  year={1998},
  volume={101 4},
  pages={
          827-33
        }
}
Pompe disease is a fatal genetic muscle disorder caused by a deficiency of acid alpha-glucosidase (GAA), a glycogen degrading lysosomal enzyme. GAA-deficient (AMD) Japanese quails exhibit progressive myopathy and cannot lift their wings, fly, or right themselves from the supine position (flip test). Six 4-wk-old acid maltase-deficient quails, with the clinical symptoms listed, were intravenously injected with 14 or 4.2 mg/kg of precursor form of recombinant human GAA or buffer alone every 2-3 d… Expand
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References

SHOWING 1-10 OF 41 REFERENCES
Japanese quail and human acid maltase deficiency: A comparative study
Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease.
Biochemical, immunological, and cell genetic studies in glycogenosis type II.
Isolation and characterisation of a recombinant, precursor form of lysosomal acid alpha-glucosidase.
Infantile and late onset form of generalised glycogenosis type ii in cattle
...
1
2
3
4
5
...