Get regular updates to your inbox.
My voice was off-pitch. I was too nervous. Though the casting directors for Singapore’s nationally televised talent show for children, “One Minute of Fame,” gave no obvious reaction, I realized the problem the second air left my lungs. Still, with all the boldness of an 8-year-old covered in makeup…
Treatment with bicalutamide and trehalose extended survival and improved motor function in a mouse model of spinal and bulbar muscular atrophy (SBMA), a study has found. Both compounds had beneficial effects on some aspects of muscle fiber structure and reduced cell death. While bicalutamide also lowered levels of…
A gentle hand shook me awake. The practice of “shaking me awake” originated with my caregiver, Danielle, who was accompanied by her 3-year-old daughter. Elena took it upon herself to wake me up. Every morning, a tiny little hand would touch my face, followed by a whispered warning.
The Tufts Medical Center, a teaching hospital of the Tufts University School of Medicine, has been awarded a Muscular Dystrophy Association (MDA) grant to investigate patient access to treatments for spinal muscular atrophy (SMA) and other diseases. The project, led by James Chambers, PhD, will evaluate access…
Life doesn’t always flow the way it’s supposed to, and by “flow,” I mean literally. I’ve recently been experiencing a lot of issues with my nasojejunal (NJ) tube. It’s become more difficult to place each time it needs to be exchanged. It clogs much more easily…
A gene-silencing therapy designed to lower the levels of two proteins was found to improve muscle strength and coordination in a mouse model of spinal and bulbar muscular atrophy (SBMA), a new study shows. The therapy, which targets the protein lysine-specific demethylase 1 (LSD1) and the protein arginine…
I have a gift inherited from my late musician mother. Simple melodies or complete orchestral scores, favorite old songs, two-piano music we played, my own original tunes, or a conglomeration of all of the above play inside my head, on demand or out of the blue. James Taylor’s…
People with spinal muscular atrophy (SMA) types 2 and 3 who received Evrysdi (ridisplam) in the SUNFISH clinical trial and its extension part continued to see improvements or stabilizations in motor function after two years, according to published trial data. Notably, patients who were originally assigned to…
Young children with spinal muscular atrophy (SMA) feel more fearful and sad, and may have a harder time recovering from distress than children who are healthy or have other conditions, a small study in India has found. These patients also seem to have more difficulty expressing their feelings…
Who would’ve thought that something as mundane as straws could become a hot topic? Yet in recent years, they’ve been featured in countless news stories, sparked debate, and been targeted by well-meaning environmental movements. I like to think I know a few things about straws. I’ve been utilizing them…
Get regular updates to your inbox.