Passion motivates us and puts a fire in our belly to strive for something outside ourselves. Without a sense of purpose, we become less motivated to rise above our current situation and struggle to find a key ingredient to a happy life. As we strive to find our way,…
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Medexus Pharmaceuticals has completed patient enrollment in its clinical trial investigating Ixinity as a preventive treatment for children younger than 12 with hemophilia B. The open-label Phase 4 trial (NCT03855280) achieved its target enrollment goal of 22 participants and is being conducted in 15 centers in Brazil,…
My oldest son, Julian, is preparing for a big audition. He hopes the casting director will hear his submission and ask him to go to New York for a final, on-site callback. If the powers that be select him, he’ll perform in his first national touring production. My son displayed…
First in a series. My story starts on a hot and humid night in June 1996 in Houston. As my wife, Cazandra, and I entered the hospital, we couldn’t help but notice the storm gathering outside. We approached the check-in counter, and I explained to the attendant that…
The first hemophilia B patient in Austria has been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to its developer, CSL Behring. “We are proud and grateful to offer hemophilia B patients in Austria a treatment option that has the potential to transform their lives,” Beate Natmessnig,…
Unexpectedly high demand for the 3,000 international units (IU) vial size of Rebinyn (nonacog beta pegol) has resulted in a temporary shortage of the Novo Nordisk factor IX (FIX) replacement therapy for those with hemophilia B, according to the company. In a statement, Novo Nordisk said…
Dear Santa, It’s that time of year again: when the lights twinkle, music fills the air, and I start making my list. But this year, I’m not wishing for things you can wrap. These wishes are for the hemophilia and bleeding disorders community — the people I love and…
The advancements in modern medicine have meant that people with hemophilia are living longer than ever, however, this brings its own set of problems for the medical profession. People living with the disease are just as likely to have other health issues when they get older as those without hemophilia,…
The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…
More than half of adults who either have hemophilia or are family members affected by the disease support the use of preconception and prenatal genetic screens, a survey reveals. However, 90 percent of this population do not agree with ending pregnancies due to hemophilia, the results also showed. Findings…