Citations 2
Medical guidelines for fibrodysplasia ossificans progressiva
Kaplan, Frederick S. , Mukaddam, Mona Al , Hidalgo‐Bravo, Alberto , Brown, Matthew A. , Cali, Amanda, Crowe, C. R., Cunto, Carmen L. De, Delai, Patricia , Diecidue, Robert J. , Friedman, Clive, Grunwald, Zvi, Haga, Nobuhiko , Kitterman, Joseph A., Levy, Charles E. , Morhart, Rolf, Netelenbos, J.C., Shore, Eileen M. , Zasloff, Michael , Pignolo, Robert J. , Al Mukaddam, Mona , Hidalgo Bravo, Alberto , Brown, Matthew , Friedlander, Lisa , Hsiao, Edward C , Shore, Eileen M , Pignolo, Robert J , Kaplan, Frederick S , Baujat, Geneviève , Cho, Tae‐Joon , Crowe, Corrie, Cunto, Carmen De , Eekhoff, Elisabeth M. W. , Hsiao, Edward C. , Keen, Richard , Madhuri, Vrisha , Scott, Christiaan , Zhang, Ke‐Qin
JBMR Plus | September 11, 2025 | Volume: 9 | Issue: 11 | Pages: No page available
Abstract Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic condition characterized by congenital malformations of the great toes and progressive heterotopic ossification (HO) in specific anatomic patterns. Present management summarized here is focused on early diagnosis, assiduous avoidance of injury and iatrogenic harm, symptomatic amelioration of painful flare-ups, and optimization of residual function. Twenty-one members of the International Clinical Council on FOP (ICC) and seven consultants from 15 countries, chosen for their clinical expertise in FOP, developed this summary statement. Further advances in therapeutics will be based on rigorous clinical trials to assess novel and emerging treatment and prevention strategies. A detailed and updated exploration of the topics outlined in this brief perspective can be found in “The Medical Management of Fibrodysplasia Ossificans Progressiva: Current Treatment Considerations” which can be found on the International Clinical Council on FOP (ICC) website (www.iccfop.org).
Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)
Hsiao, Edward C. , Rocco, Maja Di , Cali, Amanda, Zasloff, Michael , Mukaddam, Mona Al , Pignolo, Robert J. , Grunwald, Zvi, Netelenbos, Coen , Keen, Richard , Brown, Matthew A. , Cunto, Carmen De , Delai, Patricia , Haga, Nobuhiko , Morhart, Rolf, Scott, Christiaan , Zhang, Keqin , Diecidue, Robert J. , Friedman, Clive, Kaplan, Frederick S. , Eekhoff, Elisabeth M. W. , Hsiao, Edward C , Baujat, Geneviève , Cho, Tae‐Joon , Brown, Matthew , Kaplan, Frederick S
British Journal of Clinical Pharmacology | October 5, 2018 | Volume: 85 | Issue: 6 | Pages: 1199-1207
Clinical trials for orphan diseases are critical for developing effective therapies. One such condition, fibrodysplasia ossificans progressiva (FOP; MIM#135100), is characterized by progressive heterotopic ossification (HO) that leads to severe disability. Individuals with FOP are extremely sensitive to even minor traumatic events. There has been substantial recent interest in clinical trials for novel and urgently‐needed treatments for FOP. The International Clinical Council on FOP (ICC) was established in 2016 to provide consolidated and coordinated advice on the best practices for clinical care and clinical research for individuals who suffer from FOP. The Clinical Trials Committee of the ICC developed a focused list of key considerations that encompass the specific and unique needs of the FOP community – considerations that are endorsed by the entire ICC. These considerations complement established protocols for developing and executing robust clinical trials by providing a foundation for helping to ensure the safety of subjects with FOP in clinical research trials.