2024年12月17日 · Its own approvals were supported by data from the HAVEN clinical trial program, including the Phase 3 HAVEN 3 trial (NCT02847637), which found the therapy …

2017年8月24日 · Queen Victoria. Queen Victoria was a carrier of hemophilia B, a disease that she passed on to two of her daughters and one of her sons, resulting in high-profile royal cases of …

2024年6月26日 · The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults and adolescents with …

2023年12月13日 · Counseling and therapy. When seeking counseling and therapy for mental health issues, ask your hemophilia healthcare team for recommendations to mental health …

2024年7月24日 · Mim8 in hemophilia clinical trials. Early clinical trials — 4882 (NCT05127473) and FRONTIER 1 (NCT04204408) — demonstrated that Mim8 was generally safe and well …

2025年1月17日 · I don’t recall when I met Debbie Murray, but I remember the impression she left on me. Her frustration with the long and challenging journey to a proper diagnosis and …

2024年12月20日 · Researchers made an indirect comparison of data from XTEND-1 (NCT04161495) and published data for other approved factor replacement therapies, and …

2024年11月29日 · That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which Novo Nordisk’s concizumab, delivered daily under the skin for at least …

2024年10月1日 · The treatment also led to shorter hospital stays, helped patients maintain their daily living activities, and contributed to lower the doses of bypassing agents, reducing …

2025年1月3日 · No. 10 — Chinese biotech trials gene therapy for hemophilia A. Belief BioMed began the year dosing the first patient in a Phase 1/2/3 clinical trial for BBM-H803, a one-time …