Last Thursday, I visited Washington, D.C., to join my fellow blood brothers and sisters in lobbying legislators regarding issues that directly affect the bleeding disorders community. I must admit that I was a little nervous. Several times, I wondered, “What in the world am I doing here? I’m only…

There’s a pilgrimage site less than 10 minutes from my home in central New Mexico. That site, El Cerro de Tomé (Tomé Hill), is a natural landmark that’s served El Camino Real travelers for centuries. People journey to the top of the hill, 400 feet above the Rio Grande…

Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…

In our quest for security and certainty, it’s only natural to gravitate toward options that offer zero risk. Think about it: When you purchase insurance or opt for a product with a money-back guarantee, you’re essentially seeking assurance against any potential loss. This tendency to avoid risk at all…

Rare Disease Day, observed this year on Feb. 29, serves as a poignant reminder of the challenges facing those who live with uncommon medical conditions. According to the event’s website, a rare disease is defined as one that affects fewer than 1 in 2,000 individuals. Among these conditions…