Four patients taking part in a Phase 3 clinical trial of Roche’s investigational hemophilia drug emicizumab (ACE910) have experienced serious thrombotic events, blood clots that cause partial or total obstruction, Reuters reported this week. The patients were being treated for breakthrough bleeding. According to Reuters, a Roche spokesperson…
Genentech will present more than 60 abstracts at the 58th American Society of Hematology Annual Meeting (ASH 2016). Nine of Genentech’s approved or investigational medicines will be featured, including Gazyva (obinutuzumab), Rituxan (rituximab), Venclexta (venetoclax) and emicizumab, the company’s investigational drug in hemophilia. More specifically, Genentech will present…
uniQure, a company specializing in human gene therapy, will present clinical data from a Phase 1/2 dose-escalating study of AMT-060 in adults with hemophilia B at the upcoming 58th American Society of Hematology Annual Meeting (ASH 2016), to be held in San Diego, Calif., in Dec.3-6.
A recent survey has shown that the use of appropriate standardized outcome measures of hemophiliac patient treatments in routine clinical practice are not the norm. But implementing these measures could promote more effective personalized therapeutic approaches and reduce treatment-related costs. Hemophilia is a complex disease with a major impact on the…
An enzyme called activate protein C (APC), which prevents the formation of blood clots and therefore promotes bleeding could be targeted to treat hemophilia, suggests a study conducted by researcher at the University of Cambridge and the Children’s Hospital of Philadelphia.
A research team from the departments of biochemistry and pathology at the University of Pennsylvania’s School of Dental Medicine has found a successful technique using genetic engineering to coax lettuce and tobacco plants to produce foreign proteins in their leaves. The applications include producing the human clotting factor to make…
A collaborative research study offers new insights into how a protein called furin influences blood clotting and found it functions differently in hemophilia A and B, findings that are promising for improving gene therapy for hemophilia A. The study, “Circumventing furin enhances factor VIII biological activity and ameliorates bleeding…
Save One Life, the first globally focused organization to sponsor children with bleeding disorders one-on-one, recently celebrated its 15th anniversary. More than 130 guests from the hemophilia community worldwide gathered in New York City Sept. 29 at the Manhattan Penthouse on Fifth Avenue. The guest of honor was…
Recent evidence suggests that hemophilia B is clinically less severe than hemophilia A, highlighting the need to discuss further therapeutic options for each type of hemophilia. The study, “Haemophilia B is clinically less severe than haemophilia A: further evidence,” was published in Blood Transfusion. Hemophilia is an inherited, genetic…
The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of…
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