Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to investigate the pharmacokinetics, efficacy and safety of Apotransferrin replacement therapy in atransferrinemia patients.
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Hemoglobin
Timeframe: 15 year
pharmacokinetics of transferrin
Timeframe: first infusion and year 3
iron overload in organs
Timeframe: 15 years