经林业发展局批准的血友病B单剂量基因疗法将出血率减少71%。 FDA-approved single-dose gene therapy for Hemophilia B reduces bleeding episodes by 71%.

研究人员为乙型血友病研制了有希望的单剂量基因疗法,显示临床试验中出血病例减少了71%。 Researchers have developed a promising single-dose gene therapy for Hemophilia B, which has shown a 71% reduction in bleeding episodes in clinical trials. 该疗法于2024年4月获得FDA批准,使肝脏能够产生凝血因子IX,解决遗传疾病的症状. The therapy, approved by the FDA in April 2024, enables the liver to produce clotting factor IX, addressing the genetic disorder's symptoms. 这一突破可以大大提高病人的生活质量,消除对定期预防性注射的需求,从而有可能改变治疗方法。 This breakthrough could significantly enhance patients' quality of life by eliminating the need for regular prophylactic injections, potentially transforming treatment approaches.