Combining a premade SMN protein with a Spinraza-like drug (targets SMN2 gene) helped patient-derived motor neurons; potential ...
The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...
Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read ...
Four must be my unlucky number, because the worst years of my life have all ended in four: 2014, the year of major depression, and 2024, the year of suck. (I’m sure 2004 was bad, too, given that I’d ...
“Wicked” is the musical movie superstar of the moment. I loved “The Wizard of Oz” growing up, and while I haven’t seen the wildly popular adaptation on stage or screen, it’s impossible not to know at ...
Gratitude and support from family and healthcare staff can help people with spinal muscular atrophy (SMA) feel more positive, stay motivated, and plan their lives better, a study found. Programs that ...
Edward Smith, MD, is an adjunct professor of pediatrics at Duke University Hospital. He earned his medical degree from the University of Mississippi School of Medicine and has practiced since 2007. He ...
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height may help predict the risk of scoliosis in people with spinal muscular ...
If you have SMA or care for someone with the disease, you’ll know what I mean when I say we get sweaty. At first I thought it was just me, but then I read somewhere online that folks like me have ...
Swallowing function may have improved in spinal muscular atrophy (SMA) patients since disease-modifying therapies (DMT) have become available, a review study suggests. The analysis showed problems ...