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- W3087178001 endingPage "1339" @default.
- W3087178001 startingPage "1339" @default.
- W3087178001 abstract "Pompe disease, also known as glycogen storage disease type II, is caused by the lack or deficiency of a single enzyme, lysosomal acid alpha-glucosidase, leading to severe cardiac and skeletal muscle myopathy due to progressive accumulation of glycogen. The discovery that acid alpha-glucosidase resides in the lysosome gave rise to the concept of lysosomal storage diseases, and Pompe disease became the first among many monogenic diseases caused by loss of lysosomal enzyme activities. The only disease-specific treatment available for Pompe disease patients is enzyme replacement therapy (ERT) which aims to halt the natural course of the illness. Both the success and limitations of ERT provided novel insights in the pathophysiology of the disease and motivated the scientific community to develop the next generation of therapies that have already progressed to the clinic." @default.
- W3087178001 created "2020-09-25" @default.
- W3087178001 creator A5048645848 @default.
- W3087178001 creator A5061131591 @default.
- W3087178001 date "2020-09-18" @default.
- W3087178001 modified "2023-10-16" @default.
- W3087178001 title "Pompe Disease: New Developments in an Old Lysosomal Storage Disorder" @default.
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