Clinical and Translational Pharmacological Aspects of the Management of Fibrous Dysplasia of Bone

Abstract

Fibrous dysplasia (FD) is a genetic, non‐inheritable rare bone disease caused by a post‐zygotic activating mutation of the alpha subunit of the stimulatory G‐protein (Gαs) causing increased abnormal bone formation leading to pain, deformity and fractures. To date, no cure has been identified for FD/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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