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Medical research

Promising Gene Therapy Results in Treating Hemophilia B

On November 19, St. Jude Children’s Research Hospital announced the results of their ongoing gene therapy partnership with University College London and the Royal Free Hospital. The report indicates success in the trial, which used a modified adeno associated virus (AAV) 8 to deliver genetic material for making Factor IX in Hemophilia B patients. In […]


Update #2: FDA will Review Study Suggesting Higher Inhibitor Incidence in PUPS

On September 24, 2014, the medical journal, Blood, came out with a study called, Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A.  This study suggests that inhibitor incidence is higher in previously untreated patients (PUPs) with severe hemophilia A that are using Kogenate/Helixate. The Food and Drug Administration (FDA) and European Medicines Agency (EMA) have […]


Stanford University: New Form of Gene Therapy Could Cure Hemophilia in Mice

[glossary_exclude] A technique developed by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies. The ability to pop a working copy of a faulty gene into a patient’s genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine […]


FDA Approves New Treatment for Rare Form of Hemophilia

On Friday, October 24, 2014, the U.S. Food and Drug Administration approved Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A (acquired Factor VIII [FVIII] deficiency). Acquired hemophilia A is a rare, but potentially life threatening, bleeding disorder caused by the development of antibodies (immune system […]


FDA Approves First Combination Pill to Treat Hepatitis C

The U.S. Food and Drug Administration today approved Harvoni (ledipasvir and sofosbuvir) to treat chronic hepatitis C virus (HCV) genotype 1 infection. Harvoni is the first combination pill approved to treat chronic HCV genotype 1 infection. It is also the first approved regimen that does not require administration with interferon or ribavirin, two FDA-approved drugs […]

CDC_women with bleeding disorders

New Study Findings on Young Women and Bleeding Disorders

[glossary_exclude] This information was originally sent by the Division of Blood Disorders in the National Center on Birth Defects and Developmental Disabilities (NCBDDD) at the Centers for Disease Control and Prevention (CDC) in an email on Wednesday, October 8, 2014.[/glossary_exclude] ____________________________________ Information about bleeding disorders is vital for young women. It can help them recognize […]


Brighter Outlook for Community Members Affected by HCV

Hepatitis C (HCV), often referred to as the “silent killer”, has been a serious crisis for the bleeding disorders community, who contracted the disease from contaminated blood products prior to the early 1990s. Treatments exist, but with many difficult side effects and mediocre efficacy. However, advocacy in the bleeding disorders community has contributed to the […]


Update #1: Study Suggests Higher Inhibitor Incidence in PUPS Using Kogenate/Helixate

On September 24, 2014, the medical journal, Blood, came out with a study called, Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A.  This study suggests that inhibitor incidence is higher in previously untreated patients (PUPs) with severe Hemophilia A that are using Kogenate/Helixate (Bayer/Nexgen). Click here to read the […]


Important Information About Respiratory Illness EV-D68

Cooler weather and back to school triggers the start of virus season.  More time spent indoors in close proximity to those that we work, play, learn, and live with provides a great opportunity for viruses to work their way from one person to the next.  While we all try to stay healthy, it is especially important […]

Medical research

Researchers Prevent Inhibitors in Mice with Hemophilia

New and exciting news recently published in the journal Blood about a possible method to prevent and treat inhibitors. A University of Pennsylvania researcher has successfully prevented inhibitors from forming in mice with hemophilia by using a plant-based drug to teach the immune system to tolerate clotting factors. Read this University of Pennsylvania  press release in its entirety. ____________________________ […]

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