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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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Detalles Bibliográficos
Autores principales: Nikishina, Irina P., Arsenyeva, Svetlana V., Matkava, Valeria G., Arefieva, Alia N., Kaleda, Mariya I., Smirnov, Alexandr V., Blank, Leonid M., Kostik, Mikhail M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10464034/
https://www.ncbi.nlm.nih.gov/pubmed/37644581
http://dx.doi.org/10.1186/s12969-023-00856-1
Descripción
Sumario: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.