Last week, I stumbled upon the Tour de France while aimlessly flipping through TV channels. This annual professional bicycle race is highly esteemed and considered one of the most difficult events in the sport. Watching it was thrilling. The competition was captivating, and the incredible athleticism of the…

My youngest son, Caeleb, faced many health challenges with hemophilia during his early years. Throughout early elementary school, he missed more than 50 days a year. He’d go to school for a few days, and then a spontaneous internal joint bleed would take him out of school for…

European regulators have granted orphan drug status to CB 2679d, a hemophilia B therapy that Catalyst Biosciences developed to prevent bleeding episodes. The therapy, known as ISU304 in South Korea, prevents bleeding by increasing hemophilia B patients’ level of a blood protein called Factor IX, which is deficient in the disease.

Catalyst Biosciences has amended the protocol of its ongoing Phase 1/2 clinical trial investigating the highly potent recombinant human factor IX variant CB 2679d in previously treated hemophilia B patients. The amendment has shortened the study’s duration, which is a dose-escalation study to investigate the safety and drug and organism responses (pharmacokinetics and…

Note: This column shares a woman’s self-described experience administering herself factor despite a doctor’s advice. Consult your doctor before starting or stopping any therapy. For the first time, I attended the Coalition for Hemophilia B’s annual symposium, held late last month in Dallas. It was an enriching experience where…

After one year, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to safely and effectively increase factor IX (FIX) activity and prevent bleeds in men with moderate to severe hemophilia B, according to new data from the Phase 3 HOPE-B trial. The men participating in the study did…

SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…

When I traveled with HAPLOS, the Hemophilia Philippines Foundation, about six years ago to visit families with bleeding disorders in rural provinces, I expected hardship. What I didn’t expect was how drastically the same diagnosis could play out depending on where and how you lived. My husband, Jared,…

During my husband Jared’s recent hospitalization, I heard one particular sentence more times than I can count: “We’ll wait for Doc’s approval.” Jared was admitted for a burn on his hand, an injury that, on its own, often follows a fairly standard treatment path. But because he lives with…

“Be prepared.” Scouts around the world have this motto ingrained in their hearts. While I was a Girl Scout for only a short time, this practice remains essential to me. I look ahead and consider the numerous ways a situation may manifest. As the mother of two sons with…