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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: diecidue rj. 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.
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: diecidue rj. 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.
Palovarotene in fibrodysplasia ossificans progressiva: review and perspective.
Verheij VA, Diecidue RJ, Botman E, Harrington JD, Haga N, Hidalgo-Bravo A, Delai PLR, Madhuri V, Al Mukaddam M, Zhang K, Cho TJ, Morhart R, Keen R, De Cunto CL, Friedman CS, Grunwald Z, Zasloff M, Netelenbos JC, Hsiao E, Kaplan FS, Pignolo RJ, Scott C, Eekhoff EMW. Verheij VA, et al. Among authors: diecidue rj. Expert Opin Pharmacother. 2025 Jan 20. doi: 10.1080/14656566.2025.2452938. Online ahead of print. Expert Opin Pharmacother. 2025. PMID: 39834193 Review.
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