Synonyms: MZ101
Compound class:
Synthetic organic
Comment: MZ-101 is a preclinical, small molecule, selective inhibitor of glycogen synthase 1 (GYS1; P13807) [1]. It was designed as a strategy for the treatment of glycogen storage disorders such as Pompe disease, via a mechanism that reduces glycogen synthesis and storage in muscle. Inhibition of GYS1 is proposed as an alternative/or adjunct to alpha glucosidase (GAA) enzyme replacement therapy for Pompe disease.
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