ALPK1 and retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache syndrome: In a mouse model of ROSAH syndrome generated through the knock-in of heterozygous human ALPK1 harboring the p.T237M mutation, DF-003 inhibits upregulation of inflammatory cytokines in the retina, optic nerve, and cerebral cortex.