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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…
Most children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) retain motor function that exceeds what’s normally seen in untreated disease, according to a new analysis. In fact, a significant number of children who received the approved gene therapy were found to achieve…
Sobbing a blubbery and snotty cry while waiting for an elevator was likely my rock-bottom moment in January. I’d just left the interventional radiology department after another stressful procedure at the hospital. Thankfully, it went as easily as it could have, given the difficult situation. But…
Once upon a time, I was a child who grew up with fairy tales, love songs on the radio and CDs for my Walkman, young adult romance novels, and romance dramas on TV and in films. Romance was — and is — as ubiquitous as Valentine’s Day discounts for…
Some videos on YouTube, particularly in-depth ones made by healthcare professionals, provide high-quality and reliable information about treatments for spinal muscular atrophy (SMA), a new study highlights. But many of the examined videos concerning SMA treatments are neither high quality nor reliable, the researchers found, adding importance to…
The 2021 Netflix movie “Awake” imagines a world in which a mysterious catastrophe wipes out all electronics and takes away humanity’s ability to sleep. Mass hysteria ensues within hours of this apocalyptic event, as people long to let their minds and bodies rest. Citizens turn against one…
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