Persons with bleeding disorders require treatment with clotting factor concentrates for prevention and treatment of bleeding.
Clotting factor therapies are neither pharmacologically nor therapeutically equivalent and vary based upon purity, half-…
A major issue in safety of gene transfer has been assessment of long-term risks, including risks related to insertional mutagenesis and/or to unanticipated consequences, of long-term expression of the donated gene. FDA is currently recommending…
Embryonic and adult stem cell research offers the potential to cure hemophi1ia and other bleeding disorders and their complications.
The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation unanimously supports…
Individuals with bleeding disorders may require analgesics to supplement their clotting factor treatment plan. This includes individuals with hemophilia and joint pain associated with acute hemorrhages or chronic hemophilic arthropathy, as well as…
The Pennsylvania Department of Public Welfare (DPW) is considering the implementation of a "Preferred Drug List" (PDL) for clotting factor concentrates for hemophilia patients on Medicaid. This proposed policy of the DPW, which may designate only a…
Patients with hemophilia A or B who develop inhibitors to factor VIII or IX may no longer respond to clotting factor VIII or IX concentrates to prevent or control bleeding episodes.
Lack of an effective clotting factor product to prevent or treat…
In 1996 the National Hemophilia Foundation initiated an annual Gene Therapy and Novel Technologies Workshop to bring together immunologists, clinicians and researchers in gene therapy. These workshops have been invaluable in providing an open and…
The use of herbal or homeopathic products to treat bleeding disorders is increasingly being marketed to consumers, especially via the internet. In recent advertisements, two oral herbal products, Wilbrintin and Willetab, neither of which contain von…
An increasing number of individuals in the USA are developing a need for long term or indefinite oral anticoagulation. Such conditions include atrial fibrillation, mechanical heart valves, and venous thromboembolic disease.
Oral anticoagulated…
Bleeding disorders such as hemophilia are chronic disorders characterized by bleeding episodes that may occur spontaneously or after mild to severe trauma. The timing and severity of bleeding episodes are unpredictable, even for patients on…