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…

Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…

I arrived late for the concert. An usher whisked me to my seat, as my husband and our son Caeleb had already arrived. As I walked down the aisle, feeling very conspicuous in my clerical collar (I didn’t have time to change), I noticed the backs of hundreds…

A real-world study in Spain found that, among hemophilia patients, those with hemophilia A may have have worse joint health — and more joint damage, particularly in the ankles — than those with hemophilia B. Importantly, though, such damage appeared to be less frequent for individuals on regular…