Traumatic myositis ossificans circumscripta (MOC).

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Abstract

[Indexed for MEDLINE] Conflict of interest statement: Competing interests: None declared. 12. Br J Clin Pharmacol. 2019 Jun;85(6):1180-1187. doi: 10.1111/bcp.13823. Epub 2019 Jan 6. Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva. Wentworth KL(1)(2), Masharani U(1), Hsiao EC(1)(2). Author information: (1)Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine, University of California, San Francisco, CA, USA. (2)Institute for Human Genetics, University of California, San Francisco, CA, USA. Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease in which heterotopic bone forms in muscle and soft tissue, leading to joint dysfunction and significant disability. FOP is progressive and many patients are wheelchair-bound by the 3rd decade of life. FOP is caused by an activating mutation in the ACVR1 gene, which encodes the activin A Type 1 receptor. Aberrant signalling through this receptor leads to abnormal activation of the pSMAD 1/5/8 pathway and triggers the formation of bone outside of the skeleton. There is no curative therapy for FOP; however, exciting advances in novel therapies have developed recently. Here, we review the clinical and translational pharmacology of three drugs that are currently in clinical trials (palovarotene, REGN 2477 and rapamycin) as well as other emerging treatment strategies for FOP. © 2018 The British Pharmacological Society. DOI: 10.1111/bcp.13823 PMCID: PMC6533435