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Sanofi Genzyme

WORLDSymposium 2022 | Feb 7, 2022 - Feb 11, 2022

Pompe

Pompe disease (also known as acid maltase deficiency or glycogen storage disease type II) is a rare genetic lysosomal storage disease with a wide range of clinical phenotypes, presenting in infancy, childhood, or adulthood. Pompe disease is inherited in an autosomal recessive manner and caused by two pathogenic variants in the GAA gene. This leads to an absence or deficiency of the lysosomal enzyme acid α-glucosidase (GAA), essential for the degradation of glycogen. It results in progressive accumulation of lysosomal glycogen that can affect all muscle types.

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