Survival and developmental milestones among Pompe registry patients with classic infantile-onset Pompe disease with different timing of initiation of treatment with enzyme replacement therapy (ERT)


Conference Paper

Full Text

Duke Authors

Cited Authors

  • Chien, Y-H; van der Ploeg, A; Jones, SA; Byrne, B; Vellodi, A; Leslie, N; Mengel, E; Shankar, S; Tanpaiboon, P; Stockton, DW; Hennermann, JB; Devecseri, Z; Kempf, J; Keutzer, J; Kishnani, PS

Published Date

  • February 2014

Published In

Volume / Issue

  • 111 / 2

Start / End Page

  • S31 - S32

Published By

International Standard Serial Number (ISSN)

  • 1096-7192

Digital Object Identifier (DOI)

  • 10.1016/j.ymgme.2013.12.057