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19 results on '"Morhart, Rolf"'

2. Most Fractures Treated Nonoperatively in Individuals With Fibrodysplasia Ossificans Progressiva Heal With a Paucity of Flareups, Heterotopic Ossification, and Loss of Mobility

Author

Lindborg, Carter M., Al Mukaddam, Mona, Baujat, Genevieve, Cho, Tae-Joon, De Cunto, Carmen L., Delai, Patricia L. R., Eekhoff, Elisabeth M. W., Haga, Nobuhiko, Hsiao, Edward C., Morhart, Rolf, de Ruiter, Ruben, Scott, Christiaan, Seemann, Petra, Szczepanek, Małgorzata, Tabarkiewicz, Jacek, Pignolo, Robert J., and Kaplan, Frederick S.

Published
2023
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3. Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)

Author

Hsiao, Edward C, Di Rocco, Maja, Cali, Amanda, Zasloff, Michael, Mukaddam, Mona Al, Pignolo, Robert J, Grunwald, Zvi, Netelenbos, Coen, Keen, Richard, Baujat, Genevieve, Brown, Matthew A, Cho, Tae‐Joon, De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Morhart, Rolf, Scott, Christiaan, Zhang, Keqin, Diecidue, Robert J, Friedman, Clive S, Kaplan, Fredrick S, and Eekhoff, Elisabeth MW

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Clinical Trials and Supportive Activities, Rare Diseases, Bone Remodeling, Clinical Trials as Topic, Consensus, Humans, Myositis Ossificans, Ossification, Heterotopic, Patient Safety, Patient Selection, Research Design, Stakeholder Participation, Fibrodysplasia ossificans progressiva, patient safety, rare disease clinical trials, Pharmacology and Pharmaceutical Sciences, Pharmacology & Pharmacy, Pharmacology and pharmaceutical sciences
Abstract

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.

Published
2019

4. Self-reported baseline phenotypes from the International Fibrodysplasia Ossificans Progressiva (FOP) Association Global Registry

Author

Pignolo, Robert J., Cheung, Kin, Kile, Sammi, Fitzpatrick, Mary Anne, De Cunto, Carmen, Al Mukaddam, Mona, Hsiao, Edward C., Baujat, Genevieve, Delai, Patricia, Eekhoff, Elisabeth M.W., Di Rocco, Maja, Grunwald, Zvi, Haga, Nobuhiko, Keen, Richard, Levi, Benjamin, Morhart, Rolf, Scott, Christiaan, Sherman, Adam, Zhang, Keqin, and Kaplan, Fredrick S.

Published
2020
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5. The FOP Connection Registry: Design of an international patient-sponsored registry for Fibrodysplasia Ossificans Progressiva

Author

Mantick, Neal, Bachman, Eric, Baujat, Genevieve, Brown, Matt, Collins, Oliver, De Cunto, Carmen, Delai, Patricia, Eekhoff, Marelise, zum Felde, Roger, Grogan, Donna Roy, Haga, Nobuhiko, Hsiao, Edward, Kantanie, Sharon, Kaplan, Frederick, Keen, Richard, Milosevic, Jelena, Morhart, Rolf, Pignolo, Robert, Qian, Xiaobing, di Rocco, Maja, Scott, Christiaan, Sherman, Adam, Wallace, Marin, Williams, Nicky, Zhang, Keqin, and Bogard, Betsy

Published
2018
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6. Neurological symptoms in individuals with fibrodysplasia ossificans progressiva.

Author

Kitterman, Joseph A, Strober, Jonathan B, Kan, Lixin, Rocke, David M, Cali, Amanda, Peeper, Jeannie, Snow, Jennifer, Delai, Patricia LR, Morhart, Rolf, Pignolo, Robert J, Shore, Eileen M, and Kaplan, Frederick S

Subjects
Humans, Myositis Ossificans, Nervous System Diseases, Adolescent, Adult, Aged, Middle Aged, Child, Child, Preschool, Infant, Female, Male, Young Adult, Pain Research, Neurosciences, Rare Diseases, Neurological, ACVR1, Bone morphogenetic protein 4, Substance P, Clinical Sciences, Neurology & Neurosurgery
Abstract

Fibrodysplasia ossificans progressiva (FOP), a rare, disabling condition caused by gain-of-function mutations of a bone morphogenetic protein (BMP) type I receptor, leads to episodes of heterotopic ossification and resultant immobility. Neurological problems have not been associated with FOP, but neurological symptoms are commonly reported by FOP patients. To determine the prevalence of neurological symptoms and their characteristics in individuals with FOP, we conducted a survey of the 470 patient members of the International FOP Association (IFOPA) using a questionnaire about neurological symptoms. There were 168 responses (105 females, 63 males; age 1.5-68 years) from 30 countries representing 36 % of IFOPA members. Chronic neurological symptoms were reported by 86 (51 %). Prevalence of neuropathic pain (NP) was significantly increased (P < 0.001) compared to the general population, and tenfold more common in females (15 %) than males (1.6 %). Of those with NP, 94 % reported other sensory abnormalities. Prevalence of recurrent severe headaches (HA) (26 %) was similar to that in the general population, but prevalence in females with FOP (36 %) was almost fourfold greater than in males. Prevalence of NP, HA, and other sensory abnormalities was substantially higher in post-pubertal females; 33 % reported symptoms worsened during menstrual periods. Worsening of neurological symptoms during FOP flare-ups was reported by 23 %. Three patients with FOP (1.8 %) reported myoclonus, a prevalence much greater than reported in the general population (P < 0.001). Our worldwide survey indicates that neurological symptoms are common in FOP. We speculate that these symptoms are related to effects of dysregulated BMP signaling on the central and/or peripheral nervous systems.

Published
2012

9. Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

Author

Eekhoff, Elisabeth M.W., primary, de Ruiter, Ruben D., additional, Smilde, Bernard J., additional, Schoenmaker, Ton, additional, de Vries, Teun J., additional, Netelenbos, Coen, additional, Hsiao, Edward C., additional, Scott, Christiaan, additional, Haga, Nobuhiko, additional, Grunwald, Zvi, additional, De Cunto, Carmen L., additional, di Rocco, Maja, additional, Delai, Patricia L. R., additional, Diecidue, Robert J., additional, Madhuri, Vrisha, additional, Cho, Tae-Joon, additional, Morhart, Rolf, additional, Friedman, Clive S., additional, Zasloff, Michael, additional, Pals, Gerard, additional, Shim, Jae-Hyuck, additional, Gao, Guangping, additional, Kaplan, Frederick, additional, Pignolo, Robert J., additional, and Micha, Dimitra, additional

Published
2022
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10. Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

Author

Eekhoff, Elisabeth M W, de Ruiter, Ruben D, Smilde, Bernard J, Schoenmaker, Ton, de Vries, Teun J, Netelenbos, Coen, Hsiao, Edward C, Scott, Christiaan, Haga, Nobuhiko, Grunwald, Zvi, De Cunto, Carmen L, di Rocco, Maja, Delai, Patricia L R, Diecidue, Robert J, Madhuri, Vrisha, Cho, Tae-Joon, Morhart, Rolf, Friedman, Clive S, Zasloff, Michael, Pals, Gerard, Shim, Jae-Hyuck, Gao, Guangping, Kaplan, Frederick, Pignolo, Robert J, Micha, Dimitra, Eekhoff, Elisabeth M W, de Ruiter, Ruben D, Smilde, Bernard J, Schoenmaker, Ton, de Vries, Teun J, Netelenbos, Coen, Hsiao, Edward C, Scott, Christiaan, Haga, Nobuhiko, Grunwald, Zvi, De Cunto, Carmen L, di Rocco, Maja, Delai, Patricia L R, Diecidue, Robert J, Madhuri, Vrisha, Cho, Tae-Joon, Morhart, Rolf, Friedman, Clive S, Zasloff, Michael, Pals, Gerard, Shim, Jae-Hyuck, Gao, Guangping, Kaplan, Frederick, Pignolo, Robert J, and Micha, Dimitra

Abstract

Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease, in which soft connective tissue is converted into heterotopic bone through an endochondral ossification process. Patients succumb early as they gradually become trapped in a second skeleton of heterotopic bone. Although the underlying genetic defect is long known, the inherent complexity of the disease has hindered the discovery of effective preventions and treatments. New developments in the gene therapy field have motivated its consideration as an attractive therapeutic option for FOP. However, the immune system's role in FOP activation and the as-yet unknown primary causative cell, are crucial issues which must be taken into account in the therapy design. While gene therapy offers a potential therapeutic solution, more knowledge about FOP is needed to enable its optimal and safe application.

Published
2022

11. Fibrodysplasia Ossificans Progressiva: What Have We Achieved and Where Are We Now? Follow-up to the 2015 Lorentz Workshop

Author

de Ruiter, Ruben D., primary, Smilde, Bernard J., additional, Pals, Gerard, additional, Bravenboer, Nathalie, additional, Knaus, Petra, additional, Schoenmaker, Ton, additional, Botman, Esmée, additional, Sánchez-Duffhues, Gonzalo, additional, Pacifici, Maurizio, additional, Pignolo, Robert J., additional, Shore, Eileen M., additional, van Egmond, Marjolein, additional, Van Oosterwyck, Hans, additional, Kaplan, Frederick S., additional, Hsiao, Edward C., additional, Yu, Paul B., additional, Bocciardi, Renata, additional, De Cunto, Carmen Laura, additional, Longo Ribeiro Delai, Patricia, additional, de Vries, Teun J., additional, Hilderbrandt, Susanne, additional, Jaspers, Richard T., additional, Keen, Richard, additional, Koolwijk, Peter, additional, Morhart, Rolf, additional, Netelenbos, Jan C., additional, Rustemeyer, Thomas, additional, Scott, Christiaan, additional, Stockklausner, Clemens, additional, ten Dijke, Peter, additional, Triffit, James, additional, Ventura, Francesc, additional, Ravazzolo, Roberto, additional, Micha, Dimitra, additional, and Eekhoff, Elisabeth M. W., additional

Published
2021
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13. Classic and Atypical Fibrodysplasia Ossificans Progressiva (FOP) Phenotypes Are Caused by Mutations in the Bone Morphogenetic Protein (BMP) Type I Receptor ACVR1

Author

Kaplan, Frederick S., Xu, Meiqi, Seemann, Petra, Connor, J. Michael, Glaser, David L., Carroll, Liam, Delai, Patricia, Fastnacht-Urban, Elisabeth, Forman, Stephen J., Gillessen-Kaesbach, Gabriele, Hoover-Fong, Julie, Köster, Bernhard, Pauli, Richard M., Reardon, William, Zaidi, Syed-Adeel, Zasloff, Michael, Morhart, Rolf, Mundlos, Stefan, Groppe, Jay, and Shore, Eileen M.

Published
2009
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14. Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP).

Author

Di Rocco, Maja, Di Rocco, Maja, Cali, Amanda, Zasloff, Michael, Al Mukaddam, Mona, Pignolo, Robert, Grunwald, Zvi, Netelenbos, Coen, Keen, Richard, Baujat, Genevieve, Brown, Matthew, Cho, Tae-Joon, De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Morhart, Rolf, Scott, Christiaan, Zhang, Keqin, Diecidue, Robert, Friedman, Clive, Kaplan, Fredrick, Eekhoff, Elisabeth, Hsiao, Edward, Di Rocco, Maja, Di Rocco, Maja, Cali, Amanda, Zasloff, Michael, Al Mukaddam, Mona, Pignolo, Robert, Grunwald, Zvi, Netelenbos, Coen, Keen, Richard, Baujat, Genevieve, Brown, Matthew, Cho, Tae-Joon, De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Morhart, Rolf, Scott, Christiaan, Zhang, Keqin, Diecidue, Robert, Friedman, Clive, Kaplan, Fredrick, Eekhoff, Elisabeth, and Hsiao, Edward

Abstract

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.

Published
2019

15. Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)

Author

Hsiao, Edward C., primary, Di Rocco, Maja, additional, Cali, Amanda, additional, Zasloff, Michael, additional, Al Mukaddam, Mona, additional, Pignolo, Robert J., additional, Grunwald, Zvi, additional, Netelenbos, Coen, additional, Keen, Richard, additional, Baujat, Genevieve, additional, Brown, Matthew A., additional, Cho, Tae‐Joon, additional, De Cunto, Carmen, additional, Delai, Patricia, additional, Haga, Nobuhiko, additional, Morhart, Rolf, additional, Scott, Christiaan, additional, Zhang, Keqin, additional, Diecidue, Robert J., additional, Friedman, Clive S., additional, Kaplan, Fredrick S., additional, and Eekhoff, Elisabeth M.W., additional

Published
2018
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16. International physician survey on management of FOP: A modified Delphi study

Author

Di Rocco, Maja, Bertamino, Marta, Brown, Matt, De Cunto, Carmen, Delai, Patricia, Eekhoff, Elisabeth, Haga, Nobuhiko, Hsiao, Edward, Keen, Richard, Morhart, Rolf, Pignolo, Robert, Kaplan, Frederick, Di Rocco, Maja, Bertamino, Marta, Brown, Matt, De Cunto, Carmen, Delai, Patricia, Eekhoff, Elisabeth, Haga, Nobuhiko, Hsiao, Edward, Keen, Richard, Morhart, Rolf, Pignolo, Robert, and Kaplan, Frederick

Published
2017

17. International physician survey on management of FOP: a modified Delphi study

Author

Di Rocco, Maja, primary, Baujat, Genevieve, additional, Bertamino, Marta, additional, Brown, Matthew, additional, De Cunto, Carmen L., additional, Delai, Patricia L. R., additional, Eekhoff, Elisabeth M. W., additional, Haga, Nobuhiko, additional, Hsiao, Edward, additional, Keen, Richard, additional, Morhart, Rolf, additional, Pignolo, Robert J., additional, and Kaplan, Frederick S., additional

Published
2017
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18. Classic and atypical fibrodysplasia ossificans progressiva (FOP) phenotypes are caused by mutations in the bone morphogenetic protein (BMP) type I receptor ACVR1

Author

Kaplan, Frederick S., primary, Xu, Meiqi, additional, Seemann, Petra, additional, Connor, J. Michael, additional, Glaser, David L., additional, Carroll, Liam, additional, Delai, Patricia, additional, Fastnacht-Urban, Elisabeth, additional, Forman, Stephen J., additional, Gillessen-Kaesbach, Gabriele, additional, Hoover-Fong, Julie, additional, Köster, Bernhard, additional, Pauli, Richard M., additional, Reardon, William, additional, Zaidi, Syed-Adeel, additional, Zasloff, Michael, additional, Morhart, Rolf, additional, Mundlos, Stefan, additional, Groppe, Jay, additional, and Shore, Eileen M., additional

Published
2008
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19. A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva

Author

Shore, Eileen M, primary, Xu, Meiqi, additional, Feldman, George J, additional, Fenstermacher, David A, additional, Cho, Tae-Joon, additional, Choi, In Ho, additional, Connor, J Michael, additional, Delai, Patricia, additional, Glaser, David L, additional, LeMerrer, Martine, additional, Morhart, Rolf, additional, Rogers, John G, additional, Smith, Roger, additional, Triffitt, James T, additional, Urtizberea, J Andoni, additional, Zasloff, Michael, additional, Brown, Matthew A, additional, and Kaplan, Frederick S, additional

Published
2006
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