In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia.

Autor: Diogo Gonçalves, Guylène Rignol, Pierre Dellugat, Guido Hartmann, Stephanie Sarrazy Garcia, Jeffrey Stavenhagen, Luca Santarelli, Elvire Gouze, Christian Czech

Publikováno v: PLoS ONE, Vol 15, Iss 12, p e0244368 (2020)

Achondroplasia is a rare genetic disorder caused by mutations in the Fibroblast Growth Factor receptor 3 (FGFR3). These mutations lead to aberrant increase of inhibitory signaling in proliferating chondrocytes at the growth plate. Recifercept is a po

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Longitudinal Imaging of the Skull Base Synchondroses Demonstrate Prevention of a Premature Ossification After Recifercept Treatment in Mouse Model of Achondroplasia.

Autor: Rignol G; Rare Disease Unit Pfizer, Research and Development Nice France., Garcia S; Bionea Biot France., Authier F; University of Dundee Dundee UK., Smith K; Université Côte d'Azur, CNRS, Inserm, iBV Nice France., Tosello L; Université Côte d'Azur, CNRS, Inserm, iBV Nice France., Marsault R; Rare Disease Unit Pfizer, Research and Development Nice France., Dellugat P; Rare Disease Unit Pfizer, Research and Development Nice France., Goncalves D; Rare Disease Unit Pfizer, Research and Development Nice France., Brouillard M; Université Côte d'Azur, CNRS, Inserm, iBV Nice France., Stavenhagen J; Therini Bio South San Franscico CA USA., Santarelli L; VectivBio Basel Switzerland., Czech C; Rare Disease Unit Pfizer, Research and Development Nice France., Gouze E; InnoSkel Biot France.

Publikováno v: JBMR plus [JBMR Plus] 2021 Nov 09; Vol. 6 (2), pp. e10568. Date of Electronic Publication: 2021 Nov 09 (Print Publication: 2022).

Phase 1b study on the repurposing of meclizine hydrochloride for children with achondroplasia.

Autor: Matsushita, Masaki¹ (AUTHOR) masakim@med.nagoya-u.ac.jp, Kitoh, Hiroshi^2,3 (AUTHOR), Mishima, Kenichi¹ (AUTHOR), Kamiya, Yasunari¹ (AUTHOR), Kato, Daisaku¹ (AUTHOR), Takemoto, Genta¹ (AUTHOR), Sawamura, Kenta^1,2 (AUTHOR), Ueno, Shinji^4,5 (AUTHOR), Yasuhiro, Nakai⁶ (AUTHOR), Nishida, Kazuki⁶ (AUTHOR), Imagama, Shiro¹ (AUTHOR)

Publikováno v: PLoS ONE. 7/10/2023, Vol. 18 Issue 7, p1-13. 13p.

Radiological characteristics of skeletal growth in neonates and infants with achondroplasia.

Autor: Miyahara D; Department of Pediatrics, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.; Department of Pediatrics, Hiroshima City Hiroshima Citizens Hospital, Hiroshima, Japan., Hasegawa K; Department of Pediatrics, Okayama University Hospital, Okayama, Japan., Ago Y; Department of Pediatrics, Okayama University Hospital, Okayama, Japan., Futagawa N; Department of Pediatrics, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.; Department of Pediatrics, Okayama University Hospital, Okayama, Japan., Miyahara H; Department of Pediatrics, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan., Higuchi Y; Department of Pediatrics, Okayama University Hospital, Okayama, Japan., Yamada K; Department of Orthopedics, Okayama University Hospital, Okayama, Japan., Tetsunaga T; Department of Orthopedics, Okayama University Hospital, Okayama, Japan., Moriwake T; Department of Pediatrics, Iwakuni Clinical Center, National Hospital Organization, Iwakuni, Japan., Tanaka H; Department of Pediatrics, Okayama Saiseikai General Hospital, Okayama, Japan., Tsukahara H; Department of Pediatrics, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.

Publikováno v: American journal of medical genetics. Part A [Am J Med Genet A] 2024 May; Vol. 194 (5), pp. e63525. Date of Electronic Publication: 2023 Dec 29.