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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
Gustavo A Heresi1, James K Stoller
1Respiratory Institute, Cleveland Clinic, 9500 Euclid Avenue, Cleveland, Ohio 44195, USA.
Alpha-1 antitrypsin augmentation therapy can slow lung function decline in emphysema patients. This treatment is well-tolerated and may improve survival, though cost-effectiveness requires further study.
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