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Journal Article
Research Support, N.I.H., Intramural
Research Support, Non-U.S. Gov't
Review
Pompe Disease: From Basic Science to Therapy.
Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics 2018 October
Pompe disease is a rare and deadly muscle disorder. As a clinical entity, the disease has been known for over 75 years. While an optimist might be excited about the advances made during this time, a pessimist would note that we have yet to find a cure. However, both sides would agree that many findings in basic science-such as the Nobel prize-winning discoveries of glycogen metabolism, the lysosome, and autophagy-have become the foundation of our understanding of Pompe disease. The disease is a glycogen storage disorder, a lysosomal disorder, and an autophagic myopathy. In this review, we will discuss how these past discoveries have guided Pompe research and impacted recent therapeutic developments.
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