Rare Disease Report

2016 Rare Disease Hero in Lysosomal Disorders - Duke's Priya Kishnani

OCTOBER 19, 2016
Priya Kishnani, MD, PhD

In this interview during the Rare Disease Heroes Award Ceremony, Priya Kishnani, MD, PhD of Duke University Medical Center talks about the long journey to get an approved treatment for Pompe disease, as well as getting that rare disease listed on new born screening. That journey was complex with many unexpected results. But as Dr Kishnani explains, those unexpected results have led to an improved understanding of Pompe disease as well as a better understanding of our body's immunogical response to certain treatments.

Dr Kishnani's story is an inspiration to many in the rare disease community as she continues to find better treatments for Pompe disease as well as other treatments for other rare conditions such as hypophosphatasia.

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