Successful experience of tofacitinib treatment in patients with Fibrodysplasia Ossificans Progressiva

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作者
Irina P. Nikishina
Svetlana V. Arsenyeva
Valeria G. Matkava
Alia N. Arefieva
Mariya I. Kaleda
Alexandr V. Smirnov
Leonid M. Blank
Mikhail M. Kostik
机构
[1] V.A. Nasonova Research Institute of Rheumatology,
[2] Hospital Pediatry Department of Saint-Petersburg State Pediatric Medical University,undefined
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关键词
Fibrodysplasia ossificans progressiva; FOP; Heterotopic ossification; Tofacitinib; Autoinflammation; Spondyloarthritis-like disease; gene; Bone morphogenetic protein; BMP;
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摘要
Fibrodysplasia ossificans progressive (FOP) is an ultra-rare genetic disorder that is caused by a mutation in the ACVR1 gene and provokes severe heterotopic ossification. Since flares of the disease are associated with inflammation, it is assumed that JAK inhibitors can control active FOP due to blocking multiple signaling pathways.
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