In the first clinical trial of the experimental therapy SIG-001 for hemophilia A, one patient developed scarring in the spheres used to deliver the therapy that rendered the treatment ineffective, its developer, Sigilon Therapeutics, announced. The Phase 1/2 trial was put on hold by the U.S. Food and Drug…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…

My son, Caeleb, recently had his first appointment with a new dentist following our recent move, which led to our first experience with an old concern: post-traumatic stress disorder (PTSD). Caeleb, 15, has had a fear of needles for many years, and as part of his hemophilia…

A partnership between Emmes and MedGenome seeks to accelerate development of genomics-powered therapies for people with rare diseases, such as hemophilia. The companies plan to combine patient data into custom disease registries to improve research design and help with patient recruitment for clinical trials. In addition, they…

As I slowly approach the big three-zero (two more years to go!), I can’t help but reflect on the state of my health. Physical symptoms scare me now more than ever, and I want them checked as soon as possible. I’ve been trying to commit to healthier habits as well…

I cut my thumb above my knuckle a few weeks ago. It didn’t seem like a big deal, but five Band-Aids later, I began to wonder. A few hours later, I noticed my knuckle was swollen, painful, and a bit blue. My thumb wouldn’t bend, and the swelling extended down…

Advocacy is a beautiful thing. Many people find it fulfilling because it’s a way to show kindness to others while championing something close to one’s own heart. But advocacy isn’t always easy. I’ve come across some disability advocates whose dedication to the cause was tested by their experiences…

We live in a world that is loud and overwhelms us at every turn. The noise can drown out our own ability to think, leaving us frightened and unsure what our next step should be. Because I have been a caregiver to two sons with hemophilia and have to deal…

I am an advocate, born and bred. Being an advocate is simply part of my DNA. Even when I was a young girl, seeing someone be mistreated or experience prejudice angered me. The years have taught me how to fight my battles and fight for others, but when my first…

Mistakes can be costly. The damage of a single mistake can cost a few dollars or an entire livelihood. In an ideal world, we’d learn our lessons before we had the chance to make any mistakes. That way, we could avoid ever making them. It’s like going to school,…