Hemophilia News Today brought you daily coverage throughout 2021 of the latest scientific findings, treatment developments, and clinical trials related to hemophilia. As a reminder of what mattered most to you during this year, here are the top 10 most-read articles of 2021, with brief descriptions of what made…
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Marta Figueiredo, PhD, has authored numerous articles on hemophilia, covering topics such as the top hemophilia stories of 2021, the impact of mild-to-moderate hemophilia on quality of life, surgical management recommendations, and promising cell-based therapies for hemophilia A. She also reported on the effectiveness of Advate IV in preventing surgical bleeds.
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Mild-to-moderate hemophilia has a significant impact on the physical and psychosocial health, and life quality, of both men and women, according to a recent study. Findings also highlighted the unmet needs of these patient groups, supporting better guidelines and quality of care for people with mild-to-moderate disease, its researchers…
A panel of multidisciplinary experts have developed recommendations for applying best practice guidelines in the planning and managing of surgery in hemophilia patients. The guidelines were outlined in the consensus review, “Recommendations on multidisciplinary management of elective surgery in people with haemophilia,” published in the journal…
An experimental cell-based therapy using Sernova’s innovative medical device, Cell Pouch, safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and reduced bleeds in a mouse model of the disease. These are the findings of a study, “…
Continuous into-the-vein (intravenous) infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as standard, short, bolus infusions at preventing bleeds in men with moderate-to-severe hemophilia A. That finding from a Phase 3/4 clinical trial — the first controlled trial to compare the two types…
The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…
In boys and men with severe hemophilia A, joint damage accumulates with age, and can be prevented if replacement therapy is started at a younger age, according to a small, single-center study in China. In addition, poor adherence to preventive, or prophylactic treatment was significantly associated with greater…
Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…
ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing…
Roctavian (valoctocogene roxaparvovec-rvox) is an approved one-time gene therapy that’s intended to reduce the risk of bleeds in certain adults with severe hemophilia A.