I recently had the pleasure of watching the “Barbie” movie with my husband and daughter. The experience not only provided us with enjoyable family time, but also left a lasting impact on me. Beyond the vibrant visual effects, comedic gags, and engaging storyline, the movie delivered a profoundly empowering…
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Tremeau Pharmaceuticals has received guidance from the U.S. Food and Drug Administration (FDA) on how to conduct a pivotal Phase 3 trial exploring rofecoxib (TRM-201) in patients with hemophilia-associated joint pain. The advice was offered during a formal meeting with the agency, where both parties agreed on the design…
Forty-six. That’s how old I was when I finally accessed the hemophilia care that I’d needed my entire life. I believe the reason it took so long was that I’m a woman. It may seem crazy to those who aren’t familiar with the bleeding disorder. For years, the misconception…
Stimate (desmopressin acetate) was a nasal spray developed to control bleeding in people with mild hemophilia A that has been recalled and currently is unavailable. The product was sold under the brand name Octim in Europe and Octostim in Canada.
A gene therapy that can treat patients with severe hemophilia A is likely to be more cost-effective over the long term than reliance on prophylactic (preventive) therapy using factor VII (FVIII), a model-based analysis of the two treatment approaches reports. The study, “Gene therapy in hemophilia A: a cost-effectiveness…
Most of the writing I see about mental health suggests that addressing the obstacles in your way is crucial to discovering joy. While it’s important to discuss the challenges we face in the bleeding disorders community, we must also remember to celebrate and give thanks when we conquer a mountain.
The widespread adoption of telehealth during the COVID-19 pandemic has improved access to specialty care for people with hemophilia, made it more convenient, and increased patient engagement, a study based on surveys reported. Healthcare providers at an Irish care center also…
To help promote healthy lifestyle changes, the pharmaceutical company Takeda has enrolled some 900 patients with bleeding disorders like hemophilia in the Virgin Pulse Global Challenge. The annual well-being Global Challenge aims to equip teams of employees with the knowledge, tools and support necessary to enhance performance and…
Spark Therapeutics will present novel results regarding the ongoing Phase 1/2 clinical trial (NCT02484092) investigating SPK-9001 in hemophilia B patients at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, to be held July 8-13 in Berlin. Two presentations focus on data from the trial: “SPK-9001:…
Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to boost production of its AMT-060 gene therapy at its Lexington, Massachusetts, facility, in accordance with Good Manufacturing Practices guidelines.