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Pompe Disease — Gene Therapy & Clinical Trials | FollowGene

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Pompe Disease

Prevalence: 1/40000

lysosomal

Official·OMIM:232300 Official·ORPHA365

Acid maltase enzyme deficiency due to GAA gene mutations.

Associated Genes

GAAAlpha Glucosidase

Chromosome 17Autosomal Recessive

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