Prophylaxis with Idelvion (rIX‐FP) significantly improved clinical outcomes and quality of life for children with hemophilia B and eased daily life for their caregivers, Phase 3 clinical trial data show. The findings of the study, “Health-related quality of life in paediatric haemophilia B patients treated with…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…

Dear hemophilia, Truth be told, I hate you sometimes. I especially hate you when you suddenly pop out of nowhere and find ways to ruin my husband’s life. I hate you when you encroach on plans made weeks in advance; long-standing plans that were carefully and painstakingly thought out. As…

Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…

The workshop and production process over, it was curtains up for “Hemophilia: The Musical,” a unique theatrical event starring high school students affected by bleeding disorders. Performed at New World Stages in New York City on Nov. 12, the premier Broadway-style production rounded out a three-day theater and arts…

An important part of my role in the Hemophilia Association of the Philippines for Love and Service (HAPLOS) is the promotion of physical fitness and exercise. I see many young hemophiliacs strapped to wheelchairs and wearing protective gear. It pains me to see them deprived of the ability…

At 28 weeks pregnant, it seems I have entered my “nesting” phase. Two days ago, my husband Jared and I chanced upon a good deal for a crib. We had no intentions of getting a crib then, but by a stroke of serendipity, we ended up with one anyway.

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 (…

Aptevo Therapeutics provided an update on Ixinity, its hemophilia B therapy, that includes developing a larger 3,000 IU vial assay for patient convenience. The company also plans to initiate a Phase 4 clinical trial in a pediatric patients, and to apply for Ixinity’s approval outside of the United States.

I’m 25 weeks pregnant. Now that I’ve passed the halfway point of my pregnancy, I’m full of thoughts about the future. What happens when I give birth? After I give birth? When it sinks in that we are parents? Based on my ultrasound scans, it looks like we will have…