I get many opportunities to travel across the country to speak with families affected by bleeding disorders. Everywhere I go, I see familiar faces — faces that bring me great joy. There are lots of hugs, and each smile warms my heart. My talks change from event to…

Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for adults with moderately severe to severe hemophilia B. Developed by Pfizer, the gene therapy is under review by regulatory agencies in the U.S. and Europe. “Pfizer has…

uniQure is continuing to enroll participants for its global Phase 3 trial (NCT03569891) testing the potential gene therapy AMT-061 in men with severe or moderately severe hemophilia B, according to an update from the company. In addition, uniQure announced that three patients included in a Phase 2b trial (…

Recognizing risk factors of thrombosis and carefully monitoring medication doses are crucial for minimizing thrombotic complications in older patients with acquired hemophilia and serious illnesses like cardiovascular disease, a report describing four cases says. The study, “Acquired Haemophilia A. Which is the best therapeutic choice in older adults? Single…

Men with hemophilia who have a Medicaid health plan spend less on healthcare than those with employer-sponsored health insurance, due to lower reimbursement. However, Medicaid spends more on extensive healthcare users than employer-sponsored plans, according to a new study from the Centers for Disease Control and Prevention (CDC). The study,…

Organizations such as the Hemophilia Federation of America and the National Hemophilia Foundation provide the bleeding disorder community with a wide array of resources, support, and platforms to connect with others. The National Hemophilia Foundation held its annual meeting recently, and I watched as friends in the…

A single infusion of AMT-061, an investigational gene therapy for hemophilia B, continues to maintain clinically significant increases in clotting factor IX (FIX) up to six months after its administration, as demonstrated in all three patients enrolled in uniQure…

Inspired to sponsor a national conference for women with hemophilia types A and B, The Hemophilia Foundation of Michigan (HFM) became a game changer last November when it hosted an event called “Being Visible.” Gathering 103 women representing 32 states, the event featured physicians…

Ixinity remains readily available to treat people with hemophilia B despite the current COVID-19 outbreak, Medexus Pharmaceuticals has announced. The company, which acquired Ixinity from Aptevo Therapeutics, also said it will continue working to ensure continued supply throughout the pandemic. “We would like to reassure the patients…

Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, in patients with hemophilia A. Hemophilia A is an X-linked hereditary bleeding disorder that results from either reduced or absent levels of…