The growth plate is the key structure for longitudinal growth of long bones, and its function is coordinated by a complex network of systemic endocrine signals, local paracrine signals, structural support from the extracellular matrix, and intracellular signaling pathways. Dysregulation of this network can lead to short stature (SS). At present, treatment of SS mainly relies on various hormone replacement therapies, but efficacy varies. Emerging approaches, including targeted drugs, molecularly targeted delivery, and gene therapy, have demonstrated potential for clinical application. This review describes the structure and function of the growth plate, summarizes the association between its dysfunction and SS, and outlines related research and therapeutic advances, with the aim of informing studies on the molecular pathogenesis of SS and the exploration of precision treatment strategies.