Global Pompe Disease Market Driven by Increasing Developments in Gene Therapy and Enzyme Replacement Therapies

Pompe disease is a rare, progressive, autosomal recessively inherited metabolic disorder, and is often a fatal muscular disease that affects one of the lysosomal enzymes. Pompe disease can also be called as a glycogen storage disease or acid alpha-glycosidase deficiency or GAA deficiency. It mainly occurs due to accumulation of glycogen in some tissues and organs, particularly muscles, leading to abnormal functioning.

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