I am a pastor and a parent of two sons with hemophilia, a rare, genetic bleeding disorder that occurs in about one in 5,000 male births. My youngest son has a high-titer inhibitor, which means his immune system has developed antibodies to factor replacement. He’s also had an allergic reaction…

2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A. Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the…

I made it to the Manila Pen Show here in the Philippines, and I couldn’t be happier! There’s something deeply satisfying about being in a space filled with fellow enthusiasts, surrounded by exquisite writing instruments and vibrant bottles of ink. For me, collecting fountain pens is more than just a…

When my youngest son was 4, he wanted to play baseball. My nerves were shaky as I took him to register for the fall season. I hoped he would enjoy playing the sport. When the first game of the season rolled around, my wife and I sat in the bleachers,…

My husband, Jared, has fond memories of his school days when he was younger. He went to an all-boys combined elementary school and high school, where roughhousing and horsing around with friends were common. He and his classmates sometimes were crude and rowdy, he says, but they were all very…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

I’m sitting at my desk at work, focusing on projects I must accomplish before the day ends, when suddenly my ringtone fills the room with the song “Defying Gravity” from the Broadway musical “Wicked.” I see that my oldest son, Julian, is calling me. I smile when I…

I recently celebrated my 27th birthday in the company of family. Despite being unable to go out due to government-mandated COVID-19 restrictions, I still had a wonderful celebration at home. Although it was a weekday, our workload that day wasn’t too heavy or stressful. I was able to do enjoyable…

A few years ago, my family and I attended an inhibitor symposium in San Francisco. While the conference was jam-packed with incredible information, we did have a little free time to explore the city. Fisherman’s Wharf had great food and beautiful views of the harbor. We stood on a…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.