Successful experience of tofacitinib treatment in patients with Fibrodysplasia Ossificans Progressiva

Successful experience of tofacitinib treatment in patients with Fibrodysplasia Ossificans Progressiva

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 blo...

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Veröffentlicht in:Pediatric Rheumatology 2023-08, Vol.21 (1), p.1-92, Article 92
Hauptverfasser: Nikishina, Irina P, Arsenyeva, Svetlana V, Matkava, Valeria G, Arefieva, Alia N, Kaleda, Mariya I, Smirnov, Alexandr V, Blank, Leonid M, Kostik, Mikhail M
Format: Artikel
Sprache:eng
Schlagworte:
Autoinflammation
Bisphosphonates
Cytokines
Drugs
FDA approval
Fibrodysplasia ossificans progressiva
FOP
Heterotopic ossification
Kinases
Pathogenesis
Patients
Pediatrics
Physiological aspects
Range of motion
Rheumatoid arthritis
Spondyloarthritis-like disease
Steroids
Tofacitinib
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Zusammenfassung: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.
ISSN:1546-0096
1546-0096
DOI:10.1186/s12969-023-00856-1