This review examines current literature and clinical trial databases to present a comprehensive overview of a novel long-acting drug platform, transient conjugation (TransCon) technology, and its therapeutic applications in three representative rare endocrine diseases: genetic hypoparathyroidism (HP), achondroplasia (ACH), and primary growth hormone deficiency (GHD), all of which have been officially included in the Second List of Rare Diseases in China in 2023. The objective of this review is to deliver a translational perspective on TransCon-based therapies by bridging pharmacokinetic profiles with clinical efficacy, safety, and patient-reported outcomes, informing formulation development and clinical decision-making in rare endocrine diseases.
Liu, Q.Y. et al. / J. Chin. Pharm. Sci. 2026, 35 (1), 1–15.
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