Hemophilia News Today brought you daily coverage throughout 2021 of the latest scientific findings, treatment developments, and clinical trials related to hemophilia. As a reminder of what mattered most to you during this year, here are the top 10 most-read articles of 2021, with brief descriptions of what made…
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The first man with hemophilia B has been dosed in a clinical trial in China to evaluate Belief BioMed’s investigational one-time gene therapy BBM-H901. According to the company, BBM-H901 is the first investigational gene therapy given intravenously, or directly into a vein, for hemophilia B or any genetic disease…
In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia B, and this translated into fewer bleeds each year. Levels of factor IX within the normal range…
As a woman living with hemophilia, I’ve always understood that hemophilia is a man’s world. But I’ve also discovered that the men inhabiting this world are kind, compassionate superheroes with massive struggles of their own. My first National Hemophilia Foundation conference was in 2018 in Orlando, Florida. More than 2,800…
The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which…
Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…
Some years ago, when my youngest son, “MacDonald the Younger,” was about 7, he had an internal bleed into his right knee just before Thanksgiving. I was scheduled to lead a “Thanks Living” service that Tuesday, but we quickly realized we would be celebrating the holiday in a hospital…
Julian was only a baby when he received his hemophilia diagnosis, and it came as a big shock to me. I remember how much I enjoyed the newness of having a beautiful son who looked perfect. Then, the first bruise appeared. Julian has severe hemophilia A, also known as…
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
Last Sunday, I preached about a blind man named Bartimaeus. The people told the blind beggar to arise and go to Jesus, who wished to see him. It dawned on me that the people surrounding the man functioned as his advocates. Upon hearing the words of Jesus, the crowd…