In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…
Novo Nordisk reports that its treatments for hemophilia and other disorders remain available, and it is taking measures to support patients during the current COVID-19 pandemic. The company has large stock of medicines and expects to be able to meet pharmacy requests in the U.S. during the…
Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…
Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…
Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene therapy is an investigational treatment technique that uses genes or genetic material to treat or prevent…
Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer has already started recruiting participants for the lead-in Phase 3 trial (NCT03587116). which will support…
Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for hemophilia A and B. Hemophilia is caused by a lack of blood clotting factors or their faulty production,…
An interview-based study from Japan found professional support was necessary when mothers inform their daughters they might be carriers for hemophilia, as the women found themselves “not knowing what to do” and expressing “anxiety due to uncertainty.” In Japan, parents are often responsible for telling a child that a genetic…
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