A delivery method using nanotubes with a spear-like shape may open new avenues for safer, faster, and more cost-effective gene therapies for several genetic diseases, including hemophilia, according to a study. The study, “Precision-Guided Nanospears for Targeted and High-Throughput Intracellular Gene Delivery,” was recently published in the…

My family was involved in a car accident. An afternoon to the movies turned into phone calls to the police, 911, and the insurance company. Left behind by a reckless driver, I am grateful that two witnesses came forward. Having never been in an accident, I hope this is my…

As a pastor, I always remind my parishioners that it’s not enough to profess one’s faith. A person must live out the tenets of what they believe. In other words, talk is cheap. It doesn’t do a person any good to shout their love for God on Sunday and act…

Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using concizumab as a prophylactic (preventive) treatment compared with those who weren’t on any prophylaxis. That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which…

Exhaustion, stress, loneliness, and boredom are a few symptoms of living in our current world. Some people easily adapt to new ways of moving in the world, and others have a more difficult time. What is amazing is how the rare disease/disorder community seems to move forward without skipping a…

In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…

Whenever I hear the phrase, “Knowledge is power, ” I think back to the animated children’s television show Schoolhouse Rock. I loved how fun, catchy songs taught kids how bills are passed, the preamble to the Constitution, and my favorite tune, Conjunction Junction. Every episode started with an intro stating,…

During the National Conference for Women with Hemophilia and Rare Factor Deficiencies, held Oct. 3-5 in Detroit, I joined a standing-room-only session titled “More Than One Story: Exploring Joint Health in Hemophilia, Hypermobility, and Beyond.” It was refreshing to see hypermobility on the agenda — a topic that resonates…

Joint pain significantly rose and overall joint health — particularly range of motion — diminished with limitations on physical activities during the COVID-19 lockdown for people with hemophilia and joint disease, a small study from Spain reported. The study “Musculoskeletal Changes in Hemophilia Patients Subsequent to COVID−19…