Long-Term Prognosis of Patients with Infantile-Onset Pompe Disease Diagnosed by Newborn Screening (NBS) and Treated Since Birth

November 2014
Chien, Y.H., Lee, N.C., Chen, C.A., Tsai, F.J., Tsai, W.H., Shieh, J.Y., Huang, H.J., Hsu, W.C., Tsai, T.H., Hwu, W.L.

This article addresses the benefits of newborn screening for the long-term prognosis of patients with classic infantile-onset Pompe disease (IOPD).