AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), has been cleared for use under more stringent European Union rules coming into effect in a few years. Specifically, the test gained the Conformité Européenne (CE) mark under…
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Whenever I scroll through my Instagram feed, I often reflect on the “what ifs” in my life. For example, what if my life had unfolded exactly as I had envisioned it during my youth? As a child, I was a dreamer with countless aspirations. I envisioned living in a different…
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…