Correction of biochemical abnormalities and gene expression associated with improved muscle function in a phase I/II clinical trial of clenbuterol in Pompe disease patients stably treated with ERT


Conference Paper

Full Text

Duke Authors

Cited Authors

  • Koeberl, D; Case, L; Smith, EC; Li, Y; Walters, C; Hornik, C; Thurberg, B; Bali, D; Bursac, N; Kishnani, PS

Published Date

  • February 2018

Published In

Volume / Issue

  • 123 / 2

Start / End Page

  • S79 - S80

Published By

International Standard Serial Number (ISSN)

  • 1096-7192

Digital Object Identifier (DOI)

  • 10.1016/j.ymgme.2017.12.202