Clinical and translational pharmacological aspects of the management of fibrous dysplasia of bone

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Abstract

Fibrous dysplasia (FD) is a genetic, noninheritable rare bone disease caused by a postzygotic activating mutation of the α subunit of the stimulatory G-protein causing increased abnormal bone formation leading to pain, deformity and fractures. To date, no cure has been identified for FD/McCune–Albright syndrome (MAS) and treatment is symptomatic and aimed at decreasing pain and/or local bone turnover. Various drugs have been used to achieve clinical improvement in FD/MAS patients including bisphosphonates and denosumab, however further translational studies are also warranted to address unresolved pathophysiological issues and explore novel pharmacological targets for the management of FD/MAS. In this article, we review literature on the medical treatment of FD/MAS, discuss the unresolved pathophysiological issues and explore novel pharmacological targets for the management of FD/MAS.

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Rotman, M., Hamdy, N. A. T., & Appelman-Dijkstra, N. M. (2019, June 1). Clinical and translational pharmacological aspects of the management of fibrous dysplasia of bone. British Journal of Clinical Pharmacology. Blackwell Publishing Ltd. https://doi.org/10.1111/bcp.13820

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