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.
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as…
Although hemophilia, a bleeding disorder that interferes with blood clotting, is more common in men, women can have it, too.
Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for adults with moderately severe to severe hemophilia B. Developed by Pfizer, the gene therapy is under review by regulatory agencies in the U.S. and Europe. “Pfizer has…
One of the main hemophilia symptoms is excessive bleeding, which can occur into the joints. This type of bleeding can lead to joint damage, or joint arthropathy. Joint damage can limit movement and the ability to exercise for people living with this bleeding disorder.
Hemophilia News Today brought you coverage of the latest developments in research and advances in treatments for hemophilia in 2023. Here we’ve compiled a list of the top 10 most-read articles published on our site last year. We hope to continue being a resource for the hemophilia community…
Embracing the start of 2024, I reflect on the friendships I’ve made within the hemophilia community over the past year. As a woman navigating the complexities of hemophilia B and von Willebrand disease, these connections have become a source of strength and solidarity for me. Each person…
Scientists in Taiwan have created a new point-of-care device to diagnose hemophilia type A and determine a person’s blood type using just a small sample of blood. The device “is especially suitable for usage in emergency or natural disasters to provide quantitative testing in rescue and relief operations,” the…
Hemophilia joint health care involves treatments, physiotherapy, and lifestyle adjustments such as doing low-impact exercises, following a well-balanced nutrition plan, and maintaining good mental health.
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