Citing Soleo Health’s clinical expertise and data-reporting capabilities, U.S. national insurer Highmark has tapped the specialty pharmacy and infusion services provider to help care for its members who have hemophilia. An independent licensee of the Blue Cross Blue Shield Association, Highmark covers about 190 hemophilia patients.
Search results for:
Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…
Catalyst Biosciences has begun enrolling patients in a Phase 2/3 clinical trial of marzeptacog alfa (activated) as a preventive treatment for hemophilia A or B. Some patients develop immune responses — or inhibitors — to clotting factor treatments, preventing the therapies from being able to stop bleeding. Catalyst’s therapy, also known…
News
BioMarin’s Gene Therapy for Hemophilia A Continues to Prevent Bleeds After Four Years, Trial Shows
BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…
Understanding hemophilia treatment options if you have inhibitors
Fact-Checked By Lindsey Shapiro, PhD
Hemophilia inhibitors can reduce treatment effectiveness and raise bleeding risks, but several therapies can help maintain control. Understanding your options can support more confident, informed care decisions.
Beqvez (fidanacogene elaparvovec-dzkt), a discontinued gene therapy for hemophilia B, was shown to help reduce joint bleeding in Phase 3 clinical testing. That’s according to new analysis results from an ongoing global study that assessed the treatment’s effectiveness and found positive results. Specifically, “through 15 months, 12 of…
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…
Some of my most enjoyable moments are guy times with my boys. We laugh, we attempt to keep the house in order, and we succeed, despite ourselves. My sons, who have my blood running through their veins, remind me that life will be OK; that any problem can be solved,…
Hemophilia is best known for symptoms such as prolonged bleeding after an injury, easy bruising, and spontaneous bleeding into joints or muscles. Over time, repeated bleeding can also lead to less common complications. One of the rarest is the development of hemophilic pseudotumors, or benign masses caused by…
The benefits of a one-time gene therapy were sustained for up to 13 years among men with severe hemophilia B with no new safety concerns emerging, according to newly published results from a clinical trial. The findings, which encompass the longest ever follow-up for a gene therapy study in…