Article
Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP).
BMC musculoskeletal disorders - 1 Jun 2022
Smilde Bernard J, Stockklausner Clemens, Keen Richard, Whittaker Andrew, Bullock Alex N, von Delft Annette, van Schoor Natasja M, Yu Paul B, Eekhoff E Marelise W
Abstract excerpt
BACKGROUND: Fibrodysplasia Ossificans Progressiva (FOP) is a genetic, progressive and devastating disease characterized by severe heterotopic ossification (HO), loss of mobility and early death. There are no FDA approved medications. The STOPFOP team identified AZD0530 (saracatinib) as a potent inhibitor of the ALK2/ACVR1-kinase which is the causative gene for this rare bone disease. AZD0530 was proven to prevent...
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