News

Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…

Four people in a region of Northern Italy were diagnosed with acquired hemophilia A (AHA) weeks after receiving the second dose of a vaccine against COVID-19, scientists reported. The unusually high number of newly identified acquired hemophilia A cases within eight months in the Italian province led the investigators to suspect…

Hemophilia B patients living in urban mainland China have longer hospitalizations and significantly higher medical costs — including more expensive fees for coagulation factors concentrates — compared with patients with hemophilia A, a retrospective study shows. In fact, people with hemophilia B had inpatient medical costs more than…

Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A Window…

People whose hemophilia A is mild or moderate can show marked alterations to their joints on MRI scans, particularly in the ankles, despite having few joint bleeds, a study reported. These findings highlight the need for closer monitoring of joint health and a likely need for preventive treatment approaches in patients…

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that burden,…

The vast majority of women who have a bleeding disorder or are hemophilia carriers experience noticeable symptoms, such as bruising or heavy periods, a study based on survey results reported. These women also often mentioned encountering healthcare professionals who were dismissive or lacking in empathy. “Many of the experiences…

People nationwide are marking Bleeding Disorders Awareness Month, set aside each March, to call attention to disorders such as hemophilia and von Willebrand disease, and the more than 3 million U.S. residents thought to be living with them. Across the U.S., patients, caregivers, and activists are running and walking…

CSL Plasma is donating $125,000 to the American Red Cross in an effort to help lessen the burden of natural disasters to local communities in the U.S., including areas that affect plasma donors and employees involved in the manufacturing of plasma-derived therapies. Plasma protein therapies are one of the…

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…