After I had spent a few days lobbying legislators in Washington, D.C., my youngest son, Caeleb, joined me for some much-needed time together. I met him at the airport to start our madcap adventures. He was in a wheelchair due to chronic pain from numerous spontaneous bleeding…

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…

As the season of love and romance approaches, my husband, Jared, and I are already brainstorming ideas for possible February dates. We’re no strangers to unconventional coupledom. A trip to a park or a museum trumps a stroll at the mall; a night filled with laughter while watching a comedy…

Teens and young adults with moderate and severe hemophilia A on continuous preventive treatment show levels of physical activity similar to young people without the disease, but less than half of teenagers met the weekly physical activity levels recommended by the World Health organization (WHO), a study reports. “We…

Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…

A columnist reflects on how a lifelong friendship taught her the value of “meeting halfway,” a lesson that later guided her advocacy while raising a son with severe hemophilia. When gaps in care emerged, collaboration and persistence became essential to securing the support he needed.

Officials of the U.S. Food and Drug Administration and Alnylam Pharmaceuticals have agreed on safety measures and a “risk mitigation strategy” to allow the resumption of clinical studies of fitusiran, the company said in a recent press release. All ongoing trials of fitusiran had been suspended after the Sept. 7 death…

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…

Centessa Pharmaceuticals is discontinuing the clinical development of SerpinPC, its experimental under-the-skin therapy for people with hemophilia A and hemophilia B with or without inhibitors. The therapy was being evaluated in several Phase 2 clinical trials. The decision follows the recent approval of Pfizer’s Hympavzi…

Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the activity of clotting factor VIII (FVIII) are continuing in all five severe hemophilia A patients treated at the highest dose in the Alta Phase 1/2 trial. None of these…