Rare Disease Report

Pompe Disease Treatment - We Are Not Finished Yet

NOVEMBER 03, 2016
Priya Kishnani, MD, PhD



Priya Kishnani, MD, PhD of Duke University Medical Center was instrumental in getting an orphan drug approved for Pompe disease and having that disease part of new born screening. But the management of Pompe disease requires a multidisciplinary approach and enzyme replacement therapy is only one part of that care. Working closely with the patient and the clinical team is essential to improving the management of these patients.

At the same time, more treatments are needed to improve how these patients are managed. Dr Kishnani envisions second generation treatments available for Pompe disease in the near future.

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