Myostatin Research: From Molecular Understanding to Clinical Translation for Musculoskeletal and Metabolic Disorders

Myostatin (Mstn), a well-characterized member of the transforming growth factor-β (TGF-β) superfamily, serves as a key negative regulator of skeletal muscle mass. Its overactivation is closely associated with the pathogenesis of various musculoskeletal and metabolic disorders. Over the past decades, inhibiting Mstn has emerged as a promising therapeutic strategy to promote muscle growth. A range of Mstn-targeted inhibitors has been developed, yielding encouraging preclinical and clinical outcomes. These include small molecules, monoclonal antibodies, peptibodies, and gene therapy-based approaches. This review summarizes the biological structure and function of Mstn, provides a comprehensive overview of recent advances in Mstn-targeted therapeutics, and offers critical insights into future directions for drug development and clinical translation.