The Evolution of Hemophilia Therapeutics: An Illustrated Review.

María G. Español, Jessica N. Mistretta,Michael D. Tarantino,Jonathan C. Roberts

Research and Practice in Thrombosis and Haemostasis(2024)

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摘要
Hemophilia is a rare genetic bleeding disorder historically associated with high morbidity and mortality. Some individuals with hemophilia suffer associated chronic joint disease, chronic pain, and other physical and mental health challenges. In the last 50 years, a better understanding of the pathophysiology of the disease has resulted in extraordinary therapeutic advances leading to enhanced quality of life and increased life expectancy.
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关键词
Hemophilia,treatment,illustrated review,bleeding disorder,gene therapy,hemophilia treatment
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