Reversal of cardiac dysfunction after enzyme replacement in patients with infantile-onset Pompe disease.

@article{Chen2009ReversalOC,
  title={Reversal of cardiac dysfunction after enzyme replacement in patients with infantile-onset Pompe disease.},
  author={Lei-Ru Chen and Chunlu Chen and Shuenn-Nan Chiu and Yin-Hsiu Chien and Ni-Chung Lee and Ming-Tai Lin and Wuh-Liang Hwu and Jou-kou Wang and M H Wu},
  journal={The Journal of pediatrics},
  year={2009},
  volume={155 2},
  pages={
          271-5.e2
        }
}
OBJECTIVE To compare the effects of enzyme replacement therapy (ERT) on cardiac performance in symptomatic and symptom-free infants with Pompe disease. STUDY DESIGN Patients diagnosed between 1983 and 2008 were identified. Before the initiation of ERT, systolic dysfunction appeared only in patients > or = 5 months; thus we used this cut-point in age to divide clinically symptomatic patients into early and late treatment groups (Clin-E and Clin-L). Newborn screening (NBS) identified symptom… CONTINUE READING

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