المؤلفون: Pignolo, Robert J., Hsiao, Edward C., Al Mukaddam, Mona, Baujat, Geneviève, Berglund, Staffan K., Brown, Matthew A., Cheung, Angela M., De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Kannu, Peter, Keen, Richard, Le Quan Sang, Kim-Hanh, Mancilla, Edna E., Marino, Rose, Strahs, Andrew, Kaplan, Frederick S.

المصدر: Journal of Bone and Mineral Research. 38(3):381-394

مصطلحات موضوعية: clinical trial, fibrodysplasia ossificans progressiva, therapeutics

وصف الملف: electronic

الوصول الحر: https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-204678Test
https://doi.org/10.1002/jbmr.4762Test
https://umu.diva-portal.org/smash/get/diva2:1735905/FULLTEXT02.pdfTest

المؤلفون: 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

المساهمون: Horae Gene Therapy Center, Li Weibo Institute for Rare Diseases Research, Medicine, Microbiology and Physiological Systems

المصدر: Human gene therapy ; 33 ; 15-16 ; 782 ; 788 ; United States

مصطلحات موضوعية: ALK2 mutation, RNA, fibrodysplasia ossificans progressiva, gene therapy, heterotopic ossification

العلاقة: Human Gene Therapy; https://doi.org/10.1089/hum.2022.023Test; Eekhoff EMW, de Ruiter RD, Smilde BJ, Schoenmaker T, de Vries TJ, Netelenbos C, Hsiao EC, Scott C, Haga N, Grunwald Z, De Cunto CL, di Rocco M, Delai PLR, Diecidue RJ, Madhuri V, Cho TJ, Morhart R, Friedman CS, Zasloff M, Pals G, Shim JH, Gao G, Kaplan F, Pignolo RJ, Micha D. Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles. Hum Gene Ther. 2022 Aug;33(15-16):782-788. doi:10.1089/hum.2022.023. PMID: 35502479; PMCID: PMC9419966.; http://hdl.handle.net/20.500.14038/52933Test

الإتاحة: https://doi.org/10.1089/hum.2022.023Test
https://doi.org/20.500.14038/52933Test
https://hdl.handle.net/20.500.14038/52933Test

المؤلفون: Rocco, Maja Di, J Pignolo, Robert, Keen, Richard, Srinivasan, Dushyanth, J Mellis, Scott, Davis, Michelle, Orcel, Philippe, Roux, Christian, Szczepanek, Małgorzata, Bachiller-Corral, Javier, M Cheung, Angela, M Dahir, Kathryn, Mukaddam, Mona Al, Boyapati, Anita, Mohammadi, Kusha, N Economides, Aris, J Sanchez, Robert, Trotter, Dinko Gonzalez, Rhee, Susan, A Herman, Gary, DelGizzi, Richard, D Yancopoulos, George, W Eekhoff, E Marelise, S Kaplan, Frederick

المصدر: Future Rare Diseases ; volume 4, issue 1 ; ISSN 2399-5270

الإتاحة: https://doi.org/10.2217/frd-2023-0028Test

المؤلفون: Jacobson, Robert M., Pignolo, Robert J., Lazaridis, Konstantinos N.

المساهمون: National Institutes of Health, National Institute on Aging, National Cancer Institute, National Center for Advancing Translational Sciences

المصدر: Mayo Clinic Proceedings ; volume 99, issue 2, page 318-335 ; ISSN 0025-6196

مصطلحات موضوعية: General Medicine

الإتاحة: https://doi.org/10.1016/j.mayocp.2023.03.003Test
https://api.elsevier.com/content/article/PII:S0025619623001167?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S0025619623001167?httpAccept=text/plainTest

المؤلفون: Warner, Sarah E, Kaplan, Frederick S, Pignolo, Robert J, Smith, Stacy E, Hsiao, Edward C, De Cunto, Carmen, Di Rocco, Maja, Harnett, Kathleen, Grogan, Donna, Genant, Harry K

المصدر: Calcified Tissue International. 109(6)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Biomedical Imaging, Rare Diseases, Detection, screening and diagnosis, 4.2 Evaluation of markers and technologies, Absorptiometry, Photon, Adult, Disease Progression, Humans, Myositis Ossificans, Ossification, Heterotopic, Tomography, X-Ray Computed, Fibrodysplasia ossificans progressiva, Whole-body computed tomography, Dual energy X-ray absorptiometry, Heterotopic ossification, Biochemistry and Cell Biology, Biomedical Engineering, Endocrinology & Metabolism, Clinical sciences, Biomedical engineering

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/0sk1j107Test

المؤلفون: Pignolo, Robert J., Hsiao, Edward C., Mukaddam, Mona Al, Baujat, Geneviève, Berglund, Staffan K., Brown, Matthew A., Cheung, Angela M., De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Kannu, Peter, Keen, Richard, Quan Sang, Kim-Hanh Le, Mancilla, Edna E., Marino, Rose, Strahs, Andrew, Kaplan, Frederick S.

المصدر: Future Rare Diseases. 3(1)

مصطلحات موضوعية: fibrodysplasia ossificans progressive, palovarotene, plain language summary, rare disease

وصف الملف: electronic

الوصول الحر: https://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-214073Test
https://doi.org/10.2217/frd-2022-0015Test
https://umu.diva-portal.org/smash/get/diva2:1794197/FULLTEXT01.pdfTest

المؤلفون: 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, Eekhoff, Elisabeth MW

المصدر: British Journal of Clinical Pharmacology. 85(6)

مصطلحات موضوعية: 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

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/2k61w6nzTest

المؤلفون: di Rocco, Maja, Forleo-Neto, Eduardo, Pignolo, Robert J., Keen, Richard, Orcel, Philippe, Funck-Brentano, Thomas, Roux, Christian, Kolta, Sami, Madeo, Annalisa, Bubbear, Judith S., Tabarkiewicz, Jacek, Szczepanek, Małgorzata, Bachiller-Corral, Javier, Cheung, Angela M., Dahir, Kathryn M., Botman, Esmée, Raijmakers, Pieter G., Mukaddam, Mona Al, Tile, Lianne, Portal-Celhay, Cynthia, Sarkar, Neena, Hou, Peijie, Musser, Bret J., Boyapati, Anita, Mohammadi, Kusha, Mellis, Scott J., Rankin, Andrew J., Economides, Aris N., Trotter, Dinko Gonzalez, Herman, Gary A., O’Meara, Sarah J., DelGizzi, Richard, Weinreich, David M., Yancopoulos, George D., Eekhoff, E. Marelise W., Kaplan, Frederick S.

المصدر: di Rocco , M , Forleo-Neto , E , Pignolo , R J , Keen , R , Orcel , P , Funck-Brentano , T , Roux , C , Kolta , S , Madeo , A , Bubbear , J S , Tabarkiewicz , J , Szczepanek , M , Bachiller-Corral , J , Cheung , A M , Dahir , K M , Botman , E , Raijmakers , P G , Mukaddam , M A , Tile , L , Portal-Celhay , C , Sarkar , N , Hou , P , Musser , B J ....

العلاقة: https://research.vumc.nl/en/publications/17c58423-416d-4035-93e4-5aa4b74c387bTest

الإتاحة: https://doi.org/10.1038/s41591-023-02561-8Test
https://research.vumc.nl/en/publications/17c58423-416d-4035-93e4-5aa4b74c387bTest
http://www.scopus.com/inward/record.url?scp=85172773623&partnerID=8YFLogxKTest

المؤلفون: Pignolo, Robert J., Al Mukaddam, Mona, Baujat, Geneviève, Brown, Matthew A., De Cunto, Carmen, Hsiao, Edward C., Keen, Richard, Le Quan Sang, Kim-Hanh, Grogan, Donna R., Marino, Rose, Strahs, Andrew R., Kaplan, Frederick S.

المصدر: BMC Medical Research Methodology ; volume 23, issue 1 ; ISSN 1471-2288

مصطلحات موضوعية: Health Informatics, Epidemiology

الإتاحة: https://doi.org/10.1186/s12874-023-02080-7Test

المؤلفون: Wi, Chung-Il, King, Katherine S., Ryu, Euijung, Natoli, Traci L., Miller, Ryan P., Spiten, Matthew J., Borah, Bijan J., Takahashi, Paul Y., Yao, Xiaoxi, Noseworthy, Peter A., Pignolo, Robert J., Juhn, Young J.

المساهمون: GlaxoSmithKline Biologicals

المصدر: Journal of Primary Care & Community Health ; volume 14 ; ISSN 2150-1319 2150-1327

مصطلحات موضوعية: Public Health, Environmental and Occupational Health, Community and Home Care

الإتاحة: https://doi.org/10.1177/21501319231194967Test