Related Experiment Video

Updated: Jun 28, 2026

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

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

Laura E Case1, Dwight D Koeberl, Sarah P Young

  • 1Division of Physical Therapy, Department of Community and Family Medicine, Duke University Medical Center, Durham, North Carolina, NC 27708, USA. case0001@mc.duke.edu

Molecular Genetics and Metabolism
|October 22, 2008
PubMed

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