Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.

Journal Article

Benefits of enzyme replacement therapy with Myozyme (alglucosidase alfa), anecdotally reported in late-onset Pompe disease, range from motor and pulmonary improvement in less severely affected patients, to stabilization with minimal improvement in those with advanced disease. We report a case of a 63-year-old patient with significant morbidity who made notable motor and pulmonary function gains after two years on therapy. Thus, improvements in those with advanced disease may be possible after long-term treatment.

Full Text

Duke Authors

Cited Authors

  • Case, LE; Koeberl, DD; Young, SP; Bali, D; DeArmey, SM; Mackey, J; Kishnani, PS

Published Date

  • December 2008

Published In

Volume / Issue

  • 95 / 4

Start / End Page

  • 233 - 235

PubMed ID

  • 18930676

Pubmed Central ID

  • 18930676

Electronic International Standard Serial Number (EISSN)

  • 1096-7206

Digital Object Identifier (DOI)

  • 10.1016/j.ymgme.2008.09.001


  • eng

Conference Location

  • United States