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METHOD FOR TREATING OR PREVENTING IRON OVERLOAD IN A PATIENT HAVING THALASSEMIA
METHOD FOR TREATING OR PREVENTING IRON OVERLOAD IN A PATIENT HAVING THALASSEMIA
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机译:治疗或预防地中海贫血患者铁超负荷的方法
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摘要
The present invention relates to methods for treating or preventing iron overload or cardiomyopathy caused by iron overload in a patient having thalassemia, comprising administering to the patient a polypeptide comprising an amino acid sequence that is at least 90% identical to amino acids 29-109 of the sequence SEQ ID NO: 1, and wherein the polypeptide comprises an acidic amino acid residue at the position corresponding to position 79 of SEQ ID NO: 1 and inhibits signaling by myostatin and/or GDF11 in a cell-based assay.
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机译:本发明涉及在患有地中海贫血的患者中治疗或预防由铁超负荷引起的铁超负荷或心肌病的方法,其包括向所述患者施用包含与氨基酸的29-109位氨基酸至少90%相同的氨基酸序列的多肽。序列SEQ ID NO:1,其中所述多肽在对应于SEQ ID NO:1的79位的位置上包含酸性氨基酸残基,并且在基于细胞的测定中抑制肌生长抑制素和/或GDF11的信号传导。
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