Expanding therapeutic options for Pompe disease: a new small molecule inhibitor of glycogen synthase 1 (GYS1) shows preclinical promise in Pompe disease.

Importantly, glycogen reduction over the 3-month period on ERT at a dose of 20 mg/kg every 2 weeks did not reach statistical significance; this contrasts with multiple studies, including the reports which are cited by the authors in support of their findings ( 13 - 15 ).

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