Most are designed to target rare genetic diseases, including haemophilia B, where mutation causes problems with blood clotting.
大多数药物旨在针对罕见的遗传疾病，包括 B 型血友病，这种疾病的突变会导致凝血问题。
| 单词 | Haemophilia B |
| 释义 |
Haemophilia B
原声例句
2023-37 Most are designed to target rare genetic diseases, including haemophilia B, where mutation causes problems with blood clotting. 大多数药物旨在针对罕见的遗传疾病，包括 B 型血友病，这种疾病的突变会导致凝血问题。 经济学人 Science and technology So far, the new generation of genomic medicines have been eye-wateringly expensive—a shot of Hemgenix, a haemophilia B gene-therapy, costs $3.5m, around a million dollars more than Casgevy. 到目前为止，新一代基因药物的价格令人瞠目结舌，一剂血友病 B 基因治疗药物 Hemgenix 就要花费 350 万美元，比 Casgevy 贵出约 100 万美元。
英语百科
Haemophilia BHaemophilia B (or hemophilia B) is a blood clotting disorder caused by a mutation of the factor IX gene, leading to a deficiency of factor IX. It is the second-most common form of haemophilia, rarer than haemophilia A. Haemophilia B was first recognized as a a different kind of haemophilia in 1952. It is sometimes called Christmas disease, named after Stephen Christmas, the first patient described with this disease. In addition, the first report of its identification was published in the Christmas edition of the British Medical Journal. |
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