Most common AEs were blood and lymphatic system disorders (lymphocytosis, neutropenia), hepatobiliary disorders (hyperbilirubinemia, transaminases increased, gamma glutamyl transpeptidase (GGT) increased), cardiovascular disorders (arterial hypertension), and infections (respiratory tract infections, tonsillitis, urinary tract infection). The gene discussed is GGTLC1; the disease is lymphatic system disorder.