{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T17:23:42Z","timestamp":1791566622329,"version":"4.3.4"},"reference-count":63,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2019,1,6]],"date-time":"2019-01-06T00:00:00Z","timestamp":1546732800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100009633","name":"Eunice Kennedy Shriver National Institute of Child Health and Human Development","doi-asserted-by":"publisher","award":["F32HD091025 (to KLW)"],"award-info":[{"award-number":["F32HD091025 (to KLW)"]}],"id":[{"id":"10.13039\/100009633","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"publisher","award":["R01 AR066735"],"award-info":[{"award-number":["R01 AR066735"]}],"id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Brit J Clinical Pharma"],"published-print":{"date-parts":[[2019,6]]},"abstract":"<jats:p>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\u2010bound 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.<\/jats:p>","DOI":"10.1111\/bcp.13823","type":"journal-article","created":{"date-parts":[[2018,11,30]],"date-time":"2018-11-30T22:44:40Z","timestamp":1543617880000},"page":"1180-1187","source":"Crossref","is-referenced-by-count":59,"title":["Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva"],"prefix":"10.1111","volume":"85","author":[{"given":"Kelly L.","family":"Wentworth","sequence":"first","affiliation":[{"name":"Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine University of California  San Francisco CA USA"},{"name":"Institute for Human Genetics University of California  San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Umesh","family":"Masharani","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine University of California  San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8924-106X","authenticated-orcid":false,"given":"Edward C.","family":"Hsiao","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine University of California  San Francisco CA USA"},{"name":"Institute for Human Genetics University of California  San Francisco CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,1,6]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/jbmr.2728"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0674-5"},{"key":"e_1_2_8_4_1","first-page":"243","article-title":"Age\u2010 and joint\u2010specific risk of initial heterotopic ossification in patients who have fibrodysplasia ossificans progressiva","author":"Rocke DM","year":"1994","journal-title":"Clin Orthop Relat Res"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/ng1783"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aac4358"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1510540112"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2017.06.011"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aaf1090"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2017.08.023"},{"key":"e_1_2_8_11_1","first-page":"1","article-title":"The medical management of Fibrodysplasia ossificans progressiva: current treatment considerations","volume":"4","author":"Kaplan FS","year":"2011","journal-title":"Clinc Proce Intl Clin Consort FOP"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.injury.2014.05.018"},{"key":"e_1_2_8_13_1","article-title":"The development and impact of heterotopic ossification in burns: a review of four decades of research","volume":"3","author":"Kornhaber R","year":"2017","journal-title":"Scars Burn Heal"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/jbm4.10008"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev.pharmtox.46.120604.141156"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bone.2017.08.010"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1210\/me.8.3.274"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.148.4.679"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/0470846658.ch12"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/bdrc.10010"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.200211117"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/jor.20985"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm.2334"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms19040989"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/dvg.20201"},{"key":"e_1_2_8_26_1","doi-asserted-by":"publisher","DOI":"10.1002\/jbmr.2820"},{"key":"e_1_2_8_27_1","doi-asserted-by":"publisher","DOI":"10.1183\/09031936.00161911"},{"key":"e_1_2_8_28_1","unstructured":"Clementia announces top\u2010line results from phase 2 trial of palovarotene for treatment of patients with fibrodysplasia ossificans progressiva.2016. 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