1. From the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.); the Department of Hematology, Erasmus University Medical Center, University Medical Center Rotterdam, Rotterdam (F.W.G.L.), Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht (P.V.V.), Vascular Medicine, Amsterdam University Medical Center, University of Amsterdam (M.C.), Amsterdam Cardiovascular Sciences, Pulmonary Hypertension and Thrombosis (M.C.), and uniQure Biopharma (Y.P.L....