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HCB-101, hC Bioscience’s lead candidate to overwrite certain mutations that lead to hemophilia A, can reach the liver cells of mice, where it should then be able to drive the production of a working version of the blood-clotting protein that is faulty or missing in the disease. Such…
Last April, I had the privilege of attending the Coalition for Hemophilia B’s annual symposium in Dallas for the first time. It was a transformative experience filled with educational sessions, meaningful social connections, and a shared passion for advancing care within the hemophilia B community. The event brought together…
I am a patient. I am an expert on my own health experiences. I am an educator. I am an advocate. Hemophilia is a rare disease. Being a woman with a disorder thought once to be male-only makes it complicated to navigate. I…
Preventive treatment with Esperoct (turoctocog alfa pegol) plus joint surgery significantly decreased annual joint bleeding rates, reduced pain, and improved mobility in hemophilia A patients, according to an analysis of Pathfinder trial data. “Our results therefore support the notion that patients with haemophilia A being treated with [Esperoct]…
A second opinion can bring clarity, reassurance, and confidence when making important hemophilia care decisions. Exploring another expert’s perspective may help ensure your treatment plan aligns with your needs, priorities, and long-term health goals.
Scientists successfully combined the gene-editing tool CRISPR-Cas9 and a special type of virus to deliver corrected genes to restore the function of the coagulation factor IX (FIX) gene in young mice with hemophilia B. The study, “Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9,”…
It’s been nearly five months since I became a mom to baby Cittie. In those months, my life has been transformed into something completely different. My social media feed, which used to be full of items about academics and career, is now full of articles about babies and…
Treatment for burn injuries is usually straightforward: Cool the skin, manage the wound, control pain, and let time do the rest. That’s the version most people are familiar with. When hemophilia enters the picture, though, even a “simple” burn can quietly turn into something far more complicated. To add…
A newborn in China was diagnosed with hemophilia B after a ruptured liver caused significant abdominal bleeding, according to a case report. Due to early detection, timely surgical repair, and immediate factor-replacement therapy, the infant made a full recovery. This case shows that “neonatal hepatic rupture may be…