Based on the strong preclinical data, a plasma-derived form of Hx, CSL889, is being tested in a clinical trial as a treatment option for SCD to decrease the incidence of vaso-occlusive crisis and has been given Orphan drug status by both the European Commission and the Food and Drug Administration in the USA (ClinicalTrials.gov Identifier: NCT04285827). Here, HPX is linked to Schnyder corneal dystrophy.