Given the availability of new agents to treat FD, and a growing body of evidence that enzyme-focused treatment might mitigate progression, what is the rationale for suspecting and confirming the diagnosis of FD?

Name: Given the availability of new agents to treat FD, and a growing body of evidence that enzyme-focused treatment might mitigate progression, what is the rationale for suspecting and confirming the diagnosis of FD?
Uploaded: 2020-02-18T00:00:00+00:00

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Given the availability of new agents to treat FD, and a growing body of evidence that enzyme-focused treatment might mitigate progression, what is the rationale for suspecting and confirming the diagnosis of FD? What do U.S. and European Guidelines recommend as far as timing of intervention?

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Presenter

Robert Hopkin

Associate Professor of Clinical Pediatrics
Cincinnati Children's Hospital Medical Center
Co-Director, 22Q-VCFS Center
University of Cincinnati College of Medicine
Cincinnati, OH