Article

Preclinical studies with ground germinated barley (GGB) for oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice

2023-12-28

Abstract excerpt

<h4>ABSTRACT</h4> Genetic deficiency of lysosomal acid maltase or acid α-glucosidase (GAA) results in the orphan disease known as glycogen storage disease type II or acid maltase deficiency (AMD) or Pompe disease (PD), encompassing at least four clinical subtypes of varying severity. PD results from mutations in the GAA gene and deficient GAA activity, resulting in the accumulation of glycogen in tissues (primar...

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Literature Corpus work: 88650fa0-79fe-5a05-a64f-f12559d758ec
DOI: 10.1101/2023.12.27.573457

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