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Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the investigational medicines, Factor VIIa-CTP, is being developed as a potential treatment for hemophilia. “Through this joint venture, we…
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The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. Marketed by Pfizer, BeneFIX is a lab-made version of factor IX (FIX), the blood clotting protein that…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
During times of grief and struggle, columnist Joe MacDonald turns to his faith and looks for ways the sacred and divine manifest in his life.
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Becoming educated and informed about hemophilia is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your hemophilia journey.
There is no cure for hemophilia yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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