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Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP).
Hsiao EC, Di Rocco M, Cali A, Zasloff M, Al Mukaddam M, Pignolo RJ, Grunwald Z, Netelenbos C, Keen R, Baujat G, Brown MA, Cho TJ, De Cunto C, Delai P, Haga N, Morhart R, Scott C, Zhang K, Diecidue RJ, Friedman CS, Kaplan FS, Eekhoff EMW. Hsiao EC, et al. Among authors: haga n. Br J Clin Pharmacol. 2019 Jun;85(6):1199-1207. doi: 10.1111/bcp.13777. Epub 2018 Nov 6. Br J Clin Pharmacol. 2019. PMID: 30281842 Free PMC article. Review.
International physician survey on management of FOP: a modified Delphi study.
Di Rocco M, Baujat G, Bertamino M, Brown M, De Cunto CL, Delai PLR, Eekhoff EMW, Haga N, Hsiao E, Keen R, Morhart R, Pignolo RJ, Kaplan FS. Di Rocco M, et al. Among authors: haga n. Orphanet J Rare Dis. 2017 Jun 12;12(1):110. doi: 10.1186/s13023-017-0659-4. Orphanet J Rare Dis. 2017. PMID: 28606101 Free PMC article.
The FOP Connection Registry: Design of an international patient-sponsored registry for Fibrodysplasia Ossificans Progressiva.
Mantick N, Bachman E, Baujat G, Brown M, Collins O, De Cunto C, Delai P, Eekhoff M, Zum Felde R, Grogan DR, Haga N, Hsiao E, Kantanie S, Kaplan F, Keen R, Milosevic J, Morhart R, Pignolo R, Qian X, di Rocco M, Scott C, Sherman A, Wallace M, Williams N, Zhang K, Bogard B. Mantick N, et al. Among authors: haga n. Bone. 2018 Apr;109:285-290. doi: 10.1016/j.bone.2017.08.032. Epub 2017 Sep 1. Bone. 2018. PMID: 28866367
Self-reported baseline phenotypes from the International Fibrodysplasia Ossificans Progressiva (FOP) Association Global Registry.
Pignolo RJ, Cheung K, Kile S, Fitzpatrick MA, De Cunto C, Al Mukaddam M, Hsiao EC, Baujat G, Delai P, Eekhoff EMW, Di Rocco M, Grunwald Z, Haga N, Keen R, Levi B, Morhart R, Scott C, Sherman A, Zhang K, Kaplan FS. Pignolo RJ, et al. Among authors: haga n. Bone. 2020 May;134:115274. doi: 10.1016/j.bone.2020.115274. Epub 2020 Feb 13. Bone. 2020. PMID: 32062004
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
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. Eekhoff EMW, et al. Among authors: haga n. Hum Gene Ther. 2022 Aug;33(15-16):782-788. doi: 10.1089/hum.2022.023. Hum Gene Ther. 2022. PMID: 35502479 Free PMC article. Review.
462 results