Premium
Clinical and translational pharmacological aspects of the management of fibrous dysplasia of bone
Author(s) -
Rotman Marlous,
Hamdy Neveen Agnes Therese,
AppelmanDijkstra Natasha M.
Publication year - 2019
Publication title -
british journal of clinical pharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.216
H-Index - 146
eISSN - 1365-2125
pISSN - 0306-5251
DOI - 10.1111/bcp.13820
Subject(s) - denosumab , fibrous dysplasia , medicine , pathophysiology , mccune–albright syndrome , bioinformatics , bone pain , disease , bone remodeling , deformity , pathology , surgery , osteoporosis , biology , precocious puberty , hormone
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.