NEW THERAPEUTIC APPROACHES AND THEIR READOUT

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Late-onset Pompe disease (LOPD) is caused by impaired lysosomal glycogen clearance due to acid alpha-glucosidase (GAA) deficiency. Accumulation of glycogen in skeletal muscle and diaphragm leads to loss of muscle and respiratory function. Recombinant human GAA enzyme replacement therapy (rhGAA ERT; alglucosidase alfa) is standard treatment for LOPD. ATB200-02 (NCT02675465) is a first-in-human, open-label, Phase 1/2 trial to evaluate ATB200, a next-generation rhGAA ERT, co-administered with AT2221, an oral pharmacological chaperone, in adults with LOPD.

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