Typical administration consists of a daily intravenous bolus followed by subcutaneous injections for 30 days post-TPE, continuing until ADAMTS13 activity is >20%. By inhibiting platelet incorporation into microthrombi, the drug reduces the occurrence of TTP-related deaths, disease recurrences, and other complications during the trial period compared with placebo [3]. The gene discussed is ADAMTS13; the disease is thrombotic thrombocytopenic purpura.