News

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, taking…

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Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve BeneFIX and Ixinity for additional indications both therapies received last year for the treatment of hemophilia B, according to the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…

Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies that prevent the medication from working properly — in previously untreated patients (PUPs) with hemophilia A, according to the results of a clinical trial. These findings were published in the journal Thrombosis…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Exercises to control posture could improve joint health in children with hemophilia and should form part of regular rehabilitation programs, a study suggests. The balance disturbances uncovered in the study could provide early signs of musculoskeletal disorders that could be prevented or reduced through prompt diagnosis and therapy, researchers…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…