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 disorder…
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 increasing levels…
The hemophilia team and design experts at Nationwide Children’s Hospital partnered with students from Ohio State University’s Advanced Computing Center for the Arts and Design (ACCAD) to develop a virtual reality game that aims to help the hospital’s pediatric hemophilia patients during procedures. Hemophilia patients often have to go through…
Researchers from the Republic of Korea developed a new type of needle that is able to completely prevent bleeding following syringe needle puncture. This new type of needle can be invaluable for people with blood clotting disorders like hemophilia where syringe injections can have significant side effects such as uncontrolled bleeding.
Considerable progress has been made over the last 50 years in treating and caring for people with hemophilia but more is needed, according a large-scale analysis of men with this disease in the U.S. The report concluded that continued surveillance to monitor and inform hemophilia interventions and patient outcomes is essential,…
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