N-4′-(p-Trifluoromethylphenyl)butyl-DAB is a potent agonist of lysosomal acid α-glucosidase (GAA). This compound enhances intracellular GAA activity in a dose-dependent manner, specifically in fibroblasts from Pompe disease patients harboring the M519V mutation. It serves as a valuable tool for research into enzyme replacement therapies and the cellular mechanisms underlying lysosomal storage disorders.
N-4′-(p-Trifluoromethylphenyl)butyl-DAB is a potent agonist of lysosomal acid α-glucosidase (GAA). This compound enhances intracellular GAA activity in a dose-dependent manner, specifically in fibroblasts from Pompe disease patients harboring the M519V mutation. It serves as a valuable tool for research into enzyme replacement therapies and the cellular mechanisms underlying lysosomal storage disorders.
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