Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
2023年2月22日 · Here, we report the safety and efficacy of etranacogene dezaparvovec from Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B), a phase 3 study of …
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B…
We report post-hoc 24-month efficacy and safety data from this trial to evaluate the longer-term effects of etranacogene dezaparvovec in individuals with haemophilia B. Methods: The phase …
Lancet Haematol丨HOPE-B试验：血友病B基因疗法的长期随访结果
2024年6月4日 · HOPE-B（Health Outcomes with Padua Gene; Evaluation in Haemophilia B）试验是一项3期、开放标签、单剂量、多中心试验，招募了18岁及以上的遗传性血友病B男性患 …
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B…
2024年3月1日 · The HOPE-B trial shows that etranacogene dezaparvovec can provide durable disease correction throughout 24 months after gene therapy in men with severe or moderately …
Improved Joint Health in the Phase 3 HOPE-B Trial of …
2024年11月5日 · Methods: HOPE-B is a phase 3 open-label, single-dose, multicenter study in males aged ≥18 years with severe or moderately severe HB (FIX activity ≤2% of normal). After …
350万美元一针终生治愈，FDA批准世界首款治疗B型血友病的基因 …
美国当地时间 11 月 22 日，FDA 宣布批准了世界第一款针对 B 型血友病的一次性基因疗法——Hemgenix（Etranacogene dezaparvovec），用于治疗 18 岁及以上的成人患者。 该药物 …
全球首款！B型血友病基因疗法获批上市 - 基因治疗专区 - 生物谷
2022年11月23日 · HOPE-B是一项多中心、开放标签、单臂III期临床试验，旨在评估etranacogene dezaparvovec疗法在B型血友病成人患者中的疗效与安全性。 该研究共纳入了54名B型血友病 …
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy …
2020年11月19日 · Aims: A Phase 3, Health Outcomes with Padua gene; Evaluation in Hemophilia B (HOPE-B; NCT03569891) was established to further assess efficacy and safety of …
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B…
2024年3月1日 · Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic factor IX in …
Breaking ground in haemophilia B gene therapy: insights from the HOPE-B …
2024年3月1日 · In The Lancet Haematology, Michiel Coppens and colleagues 5 report the 24-month efficacy and safety update from the phase 3 HOPE-B trial of etranacogene …