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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Real-World Migalastat Use in Fabry Disease: Comparative Insights From the Pisani and Hughes Studies
Eleonora Riccio1, Antonio Pisani1
1Department of Public Health, Chair of Nephrology, University Federico II of Naples, Naples, Italy.
Journal of Inherited Metabolic Disease
|August 12, 2025
Abstract
No abstract available in PubMed .

