Rare Disease Report

Newborn Screening for Pompe Disease - A Catalyst For Understanding a Rare Disease

FEBRUARY 19, 2017
Priya Kishnani, MD, PhD

At WORLDSymposium in San Diego, we talked with Priya Kishnani, MD, PhD about the amount of new information gained by studying patients diagnosed at birth with Pompe disease. The data is dramatically changing our understanding of Pompe disease and its management.

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