Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…
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My husband pastors the local First United Methodist Church, where I serve as a deacon. When a parent (or two) goes down the pastoral path, their children are along for the ride. Some preachers’ kids love church, and others, not so much. My youngest son, Caeleb, falls…
A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…
Nearly all medical directors at hemophilia treatment centers in the U.S. said they would consider starting preventive Hemlibra (emicizumab-KXWH) in untreated infants with hemophilia A, ages 12 months and younger, a survey study reports. Most respondents also said they’d transition from FVIII replacement therapies, used on-demand to treat…
Hemlibra (emicizumab-KXWH) safely controlled bleeds among hemophilia A patients without inhibitors over nearly five years of preventive treatment, according to long-term data pooled from two Phase 3 clinical trials. “Five years of follow-up data showed a favorable benefit-risk profile of [Hemlibra] prophylaxis,” the researchers wrote in “…
Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…
Individually tailored prophylaxis, or preventive treatment, with Jivi (damoctocog alfa pegol) nearly halved the number of annual bleeding episodes in people with severe hemophilia A who had previously been on another replacement therapy, according to a Phase 4 post-marketing study. Researchers also observed that none of the patients…
SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
Almost three years of treatment with Jivi (damoctocog alfa pegol) reduced the annual bleeding rate among a large, multinational group of previously treated hemophilia A patients, a real-world study reports. The number of patients without joint bleeds increased during the study and…
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 a…