对“帕罗伐他汀可减少青少年 FOP 小鼠的异位骨化，但表现出明显的骨骼毒性”一文的评论作出回应。
Response to comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
机构信息
Department of Molecular and Cell Biology, University of Connecticut, Storrs, United States.
出版信息
Elife. 2019 Jan 30;8:e43928. doi: 10.7554/eLife.43928.
Abstract
We respond to concerns expressed by Pacifici and Shore (2019) about a recent paper (Lees-Shepard and Goldhamer, 2018a) in which we reported that the drug palovarotene can have severe side effects in a mouse model of fibrodysplasia ossificans progressiva.
摘要
我们对 Pacifici 和 Shore（2019）对我们最近一篇论文（Lees-Shepard 和 Goldhamer，2018a）提出的担忧做出回应。在这篇论文中，我们报告了药物 palovarotene 在纤维发育不良性骨化进展的小鼠模型中可能产生严重的副作用。
相似文献
1
Response to comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
Elife. 2019 Jan 30;8:e43928. doi: 10.7554/eLife.43928.
2
Comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
Elife. 2019 Jan 30;8:e43173. doi: 10.7554/eLife.43173.
3
Palovarotene Inhibits Heterotopic Ossification and Maintains Limb Mobility and Growth in Mice With the Human ACVR1(R206H) Fibrodysplasia Ossificans Progressiva (FOP) Mutation.
J Bone Miner Res. 2016 Sep;31(9):1666-75. doi: 10.1002/jbmr.2820. Epub 2016 Mar 12.
4
Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity.
Elife. 2018 Sep 18;7:e40814. doi: 10.7554/eLife.40814.
5
Surgical management of bilateral hip fractures in a patient with fibrodysplasia ossificans progressiva treated with the RAR-γ agonist palovarotene: a case report.
BMC Musculoskelet Disord. 2020 Apr 3;21(1):204. doi: 10.1186/s12891-020-03240-2.
6
Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP): Results of a Randomized, Placebo-Controlled, Double-Blind Phase 2 Trial.
J Bone Miner Res. 2022 Oct;37(10):1891-1902. doi: 10.1002/jbmr.4655. Epub 2022 Aug 17.
7
Palovarotene: First Approval.
Drugs. 2022 Apr;82(6):711-716. doi: 10.1007/s40265-022-01709-z.
8
Reduction of New Heterotopic Ossification (HO) in the Open-Label, Phase 3 MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP).
J Bone Miner Res. 2023 Mar;38(3):381-394. doi: 10.1002/jbmr.4762. Epub 2023 Jan 25.
9
Cartilage-derived retinoic acid-sensitive protein (CD-RAP): A stage-specific biomarker of heterotopic endochondral ossification (HEO) in fibrodysplasia ossificans progressiva (FOP).
Bone. 2018 Apr;109:153-157. doi: 10.1016/j.bone.2017.09.016. Epub 2017 Sep 28.
10
Therapeutic advances for blocking heterotopic ossification in fibrodysplasia ossificans progressiva.
Br J Clin Pharmacol. 2019 Jun;85(6):1180-1187. doi: 10.1111/bcp.13823. Epub 2019 Jan 6.
引用本文的文献
1
Sex as a Critical Variable in Basic and Pre-Clinical Studies of Fibrodysplasia Ossificans Progressiva.
Biomolecules. 2024 Feb 1;14(2):177. doi: 10.3390/biom14020177.
2
Overexpression of Wild-Type ACVR1 in Fibrodysplasia Ossificans Progressiva Mice Rescues Perinatal Lethality and Inhibits Heterotopic Ossification.
J Bone Miner Res. 2022 Nov;37(11):2077-2093. doi: 10.1002/jbmr.4617. Epub 2022 Jul 3.
3
Challenges and Opportunities for Drug Repositioning in Fibrodysplasia Ossificans Progressiva.
Biomedicines. 2021 Feb 19;9(2):213. doi: 10.3390/biomedicines9020213.
本文引用的文献
1
Comment on 'Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity'.
Elife. 2019 Jan 30;8:e43173. doi: 10.7554/eLife.43173.
2
Palovarotene reduces heterotopic ossification in juvenile FOP mice but exhibits pronounced skeletal toxicity.
Elife. 2018 Sep 18;7:e40814. doi: 10.7554/eLife.40814.
3
Stem cells and heterotopic ossification: Lessons from animal models.
Bone. 2018 Apr;109:178-186. doi: 10.1016/j.bone.2018.01.029. Epub 2018 Jan 31.
4
Activin-dependent signaling in fibro/adipogenic progenitors causes fibrodysplasia ossificans progressiva.
Nat Commun. 2018 Feb 2;9(1):471. doi: 10.1038/s41467-018-02872-2.
5
Palovarotene Inhibits Heterotopic Ossification and Maintains Limb Mobility and Growth in Mice With the Human ACVR1(R206H) Fibrodysplasia Ossificans Progressiva (FOP) Mutation.
J Bone Miner Res. 2016 Sep;31(9):1666-75. doi: 10.1002/jbmr.2820. Epub 2016 Mar 12.
6
Fibrodysplasia ossificans progressiva: mechanisms and models of skeletal metamorphosis.
Dis Model Mech. 2012 Nov;5(6):756-62. doi: 10.1242/dmm.010280.
7
An Acvr1 R206H knock-in mouse has fibrodysplasia ossificans progressiva.
J Bone Miner Res. 2012 Aug;27(8):1746-56. doi: 10.1002/jbmr.1637.
8
Evolutionary divergence of platelet-derived growth factor alpha receptor signaling mechanisms.
Mol Cell Biol. 2003 Jun;23(11):4013-25. doi: 10.1128/MCB.23.11.4013-4025.2003.
9
Platelet-derived growth factor A modulates limb chondrogenesis both in vivo and in vitro.
Mech Dev. 2000 Jun;94(1-2):13-24. doi: 10.1016/s0925-4773(00)00321-x.
10
Platelet-derived growth factor-A and its receptor are expressed in separate, but adjacent cell layers of the mouse embryo.
Development. 1992 Aug;115(4):1045-58. doi: 10.1242/dev.115.4.1045.
Zotero 插件