Omaveloxolone, a therapy designed to promote anti-stress responses in cells, may help correct stress-related dysfunction in certain cells from people with spinal muscular atrophy (SMA), according to a lab-based study. The treatment works by boosting levels of proteins that help fight stress, including several in a signaling pathway called…
Discussion
Travel has always felt like freedom to me. Some of my favorite memories were made on long road trips with friends, crisscrossing the country in my van — from Maine to Florida, the Carolinas to California, Texas to Washington, and through about a dozen national parks along the way. I…
Discussion
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) helped children with spinal muscular atrophy (SMA) gain or maintain motor milestones and motor function, a real-world study in Japan found. The RESTORE patient registry (NCT04174157) is assessing the long-term safety and efficacy of Zolgensma in clinical settings across 99 locations in the…
Discussion
I recently wrote about how I’ve been in the process of transitioning from a children’s hospital to an adult hospital. It’s meant saying goodbye to the place that knew my medical history better than anyone else, that helped keep me alive through countless challenges, and that understood my…
Discussion
When I graduated from college in 2005, I received a special gift: a large whiteboard to hang on the wall. It had 38 pictures on it showing many of my adventures with SMA and other key moments I’d had over the years. Those pictures have remained on my wall…
Discussion
I don’t talk to my ex anymore. For the sake of this column, I will have to let her know about it as a courtesy. But as far as a daily choice goes, it’s not one I make anymore. Readers who’ve kept up with my column in the last few…
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