Voncento — a highly concentrated mixture of Von Willebrand factor (VWF) and clotting factor VIII (FVIII) — was seen to safely and effectively treat or prevent bleeds in children, age 12 and younger, with severe hemophilia A, according to data from a Phase 3 trial. Findings were reported in the…

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ Phase 1/2 study evaluating the investigational therapy SIG-001 in adults with severe or moderately severe hemophilia A. The move comes after a serious adverse event (SAE) occurred involving one of the three participants…

The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…

My nephew does not like needles. Actually, “hate” is probably a better word. He hates needles. This is not ideal, given that he has hemophilia. Recently, I was helping him practice IV infusions on a makeshift “arm” I created. As we talked, I told him, “You need to own hemophilia.

My older son came to visit us for the first time since March 2020. Although we talk almost every day, there is nothing like seeing him in person. I didn’t realize how much my soul craved communion with him. We laughed and talked about how good it is to have…

Several things often happen when a woman becomes a mother. The switch of motherly intuition flips on, and many moms can make predictions without reason. (That “gut” instinct usually serves us well.) The overwhelming love for our newborn baby is a connection that ignites and lasts a lifetime. This…

Complication rates for elective surgeries in people with inherited bleeding disorders like hemophilia are low in Australia, reflecting good safety in their performance despite guidelines not always being followed, a study reported. Use of a specialty hemophilia treatment center (HTC) for surgery, and use of factor replacement therapy were among guidelines sometimes…

Last Sunday, I preached a sermon about the Old Testament story of David slaying the giant Goliath. David was young and much shorter than the very tall Goliath. The odds were against David, yet through divine intervention, he did the unthinkable and took Goliath’s life with one stone. His…

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…

I gaze at the glorious sunrise as I take off in my red RAV4. On this particular trip I plan to make my way down from my home in Northern California to Los Angeles. My daughter and I will most likely push through the 12+ hour drive in…