Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…

As a former band director, I understand the importance of each musical instrument in an ensemble. The percussion keeps the rhythm steady. The low brass offers the foundation of the music, and the trumpets, flutes, and clarinets play the melodies. Each section contributes to the overall piece. Music is an…

Hemgenix (etranacogene dezaparvovec), the first and only gene therapy approved for adults with hemophilia B, has won this year’s Prix Galien USA award in the category of Best Product for Rare/Orphan Diseases. The award — the nation’s top prize for leading-edge advances in life sciences — was received…

My husband, Jared, and I have an open-door policy for our friends. We warmly welcome them, whether they need a place to stay for the night, a safe haven after a tough experience, a spot to relax, or a place to celebrate life’s victories. In our home, friends become part…

Scrabble, the classic word game that has enthralled generations, is a captivating blend of strategy, vocabulary, and skill. I began playing competitively in 2010. At best, I am a mediocre player, but I love the game and am grateful for my connection to the Scrabble community. For me, Scrabble has…

The first participant has been dosed in a Phase 2b clinical trial that’s assessing the safety and efficacy of SerpinPC in people with hemophilia B who have inhibitors, according to an announcement from its developer, Centessa Pharmaceuticals. “We are excited to be further evaluating the potential of…

I turned on the light in my son Caeleb’s room and began my morning concert of songs to wake him up. My top 10 hits (including “Good Morning Starshine,” “Rise and Shine,” and “Good Morning” from “Bear in the Big Blue House”) fuel my soul with energy, though my 17-year-old…

As a hospice chaplain, I visit patients and their families as they are in their last months and days of life. It is humbling. Whether they’re at home or in a nursing home, these patients have something in common: They are being cared for by others. Family caregivers are the…

My husband, Jared, and I have been fur parents for a while. In 2020, we welcomed our first family pet, a golden retriever named Lucky, followed by a delightful Himalayan cat we named Billy. Our home has been lively with the presence of animals for the past three years.

This year’s Hope Conference, a national educational event presented by Hope Charities for the bleeding disorders community, is set for Nov. 2-4 in Florida, with an online option as well. The annual conference, to be presented at the Baytowne Conference Center, in Miramar Beach, is designed to bring together…