Enzyre is partnering with Takeda with a goal of developing at-home assays to help people with monitoring congenital bleeding disorders, including hemophilia, and to diagnose these disorders. This partnership builds on an existing, 2019 research collaboration between these…
The National Hemophilia Foundation (NHF) is calling for nominations for its Awards of Excellence, which honor members of the bleeding disorders community. The awards are for those who have helped improve the lives of patients with bleeding diseases, including those with hemophilia, through treatment and care, advocacy,…
The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the…
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…
Activities are underway to mark Bleeding Disorders Awareness Month, set aside each March to call attention to such disorders as hemophilia and von Willebrand disease, and the more than three million U.S. residents living with them. Patients, caregivers, and activists nationwide are jumping on social media platforms,…
People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information and insufficient engagement with other patients and healthcare providers, according to a panel of U.S. experts. In a recent study, these experts proposed new…
Sanofi has reduced the dosing of its investigational treatment fitusiran in clinical trials to lower the risk of blood clots in people with hemophilia, according to a joint statement representing patient organizations worldwide. In October 2020, the company placed a voluntary dosing hold on its full clinical…
Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve BeneFIX and Ixinity for additional indications both therapies received last year for the treatment of hemophilia B, according to the National Hemophilia Foundation. Pfizer’s BeneFIX and…
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…
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