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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
A Faggiano1, R Severino, V Ramundo
1Department of Molecular and Clinical Endocrinology and Oncology, Federico II University, Naples, Italy. afaggian@unina.it
Enzyme replacement therapy (ERT) for Fabry disease (FD) can reverse subclinical hypothyroidism. Regular thyroid function screening is essential for FD patients undergoing ERT.
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