Adults with hemophilia and ankle joint damage taking part in a yearlong pilot study largely reported that two injections of hyaluronic acid, which acts to lubricate the joint, eased ankle pain and improved movement. The treatment, given six months apart in this U.K. trial, also enabled most of these people…
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AMT-061 (etranacogene dezaparvovec), an investigational gene therapy uniQure is developing to treat hemophilia B, successfully increased factor IX (FIX) activity and controlled bleeding — while markedly reducing the need for other treatments — in the Phase 3 HOPE-B clinical trial, top-line data show. These findings will be presented at…
Historically, hemophilia has been viewed as a disease that affects men and boys, which has led to significant gender bias against women with the disease in research, diagnosis, and treatment. As a result, there’s a lack of awareness and understanding of how hemophilia affects women and…
In this video shared by Colleen, learn about a mobile app from Pfizer which helps hemophilia patients track bleeds, infusions and factor supply. It also allows those living with the disease to track their daily physical activities, steps and heart rate to help…
Last in a series. Read parts one and two. After our oldest son, Julian, was born and we took him home, the first six months went smoothly. His hemophilia diagnosis came up occasionally, but it didn’t directly affect our quality of life. We later learned that those early…
Pfizer is preparing to launch its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) after securing a U.S. approval for the treatment last month. Beqvez is the second gene therapy to be approved for adults with moderate to severe hemophilia B in the U.S., following the clearance of…
The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B — in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need. PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can…
In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in the developing world to highlight the life-changing potential of adequate treatment. In honor of this year’s theme, “Sharing Knowledge Makes Us Stronger,” Bioverativ will collaborate with members of the hemophilia community…
Sigilon Therapeutics’ Afibromer SIG-003 for Long-term Treatement of Hemophilia B Advances in EU
Sigilon Therapeutics’s Afibromer SIG-003 candidate for the long-term treatment of patients with hemophilia B recently received an Advanced Therapy Medicinal Product (ATMP) designation in the European Union. The designation was granted by the Committee for Advanced Therapies of…