Rare Disease Report

Latest Information about Gene Therapy for Pompe Disease

APRIL 13, 2017
Dwight Koeberl, MD

Dwight Koeberl, MD at Duke University talks about the latest studies being done to test gene therapy for Pompe disease.
The interview took place during Duke University's Pompe disease Patient Meeting held April 8th, 2017.

What is Pompe Disease?

Pompe disease is a rare genetic neuromuscular disorder caused by an excessive buildup and storage of glycogen in the muscle cells, resulting in degenerative muscle weakness. The muscle weakness can affect many different parts of the body and cause a variety of health problems. Symptoms include difficulty walking, trouble breathing, headache, enlarged heart and failure to thrive.

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