CRISPR/Cas9 is a gene-editing technology that can be used to alter the genetic sequence of a specific gene inside a cell. Several preclinical studies have indicated that the molecular tools in CRISPR/Cas9 might be used to treat or even cure hemophilia.
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One of the major hurdles people with hemophilia face, is complying with their medication schedule — in particular, injecting themselves several times a week with clotting factor. MORE: Seven recommended books to learn more about hemophilia. Clotting factor has a relatively short half-life (the time it takes for a person’s…
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…
Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…
Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, a mouse study has found. The study shows that the transplant of cells…
The World Federation of Hemophilia states that any person, male or female, with factor levels below 40 percent has hemophilia. However, conventional medical wisdom has not caught up. Women who have abnormal bleeding should be assessed for hemophilia, particularly when they have a family history. Yet too…
As part of a new partnership focused on hemophilia treatment and management, members of insurer Highmark will receive medication exclusively from Option Care Health, Soleo Health, and the Hemophilia Center of Western Pennsylvania. The collaboration that begins in January is aimed at keeping down costs associated with…
A 32-year-old man was diagnosed with hemophilia A after a minor injury caused disproportionate right eye bleeding, with a buildup of pressure inside the eye and vision loss, a new U.S. case study reports. According to the researchers, this case demonstrates that an eye bleed can be a rare…
“As a woman with hemophilia, what situations cause you anxiety?” a friend recently asked me. What a great question! I loved that she added “as a woman” because women with hemophilia can experience stressors that men with hemophilia don’t. Many anxiety-provoking circumstances affect men and women with hemophilia…
Obizur (susoctocog alfa) is an effective and safe therapeutic option for the treatment of severe bleeding episodes in people with acquired hemophilia A, a study in Japanese patients showed. The study “supports the use of [Obizur] as a novel therapy in the clinical management of patients with [acquired…