Pompe Disease – Clinical Outcomes

In this webinar, Dr. Priya Kishnani, Professor of Pediatrics and Chief of the Division of Medical Genetics at Duke, emphasizes the necessity to identify Pompe Disease early through NBS (newborn screening). Dr. Kishnani mentions the significant benefits to beginning ERT (enzyme replacement therapy) as soon as possible and how responsiveness to therapy changes with age and severity of condition onset.

Presenter: Dr. Priya Kishnani, Professor of Pediatrics and Chief of the Division of Medical Genetics at Duke

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