Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in children with SMA who had previously been treated with gene therapy but did not respond as…
Discussion
At the beginning of the year, I felt a sense of calm because I finally had enough nurses to complete my home nursing staff. There was no pressure to find a replacement for a nurse leaving my case. That kind of calm is priceless in my life with SMA.
Mutations that cause spinal muscular atrophy (SMA) disrupt the development of spinal cord cells, leading to fewer nerve cells in early development, according to a new study in cell models. The findings highlight potential new avenues for treating SMA in its earliest stages, researchers say. Zeynep Dokuzluoglu, a graduate…
Discussion
As a college freshman pursuing a degree in special education, I assisted in two nearby elementary classrooms. The students all had moderate cognitive challenges. I remember a few even now: Roger, with his suspenders and his family’s pigs (Fred and Ethel), and Tina and Richard, adorable twins. All three had…
Genetic screening for spinal muscular atrophy (SMA) in newborns may allow treatment to begin earlier, which could help support motor development, a study suggests. This “adds to the evidence that [newborn screening] for SMA is associated with significantly earlier time to first treatment and is subsequently associated with improved…
At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases. Bionews, the parent company of this site, sat down with John F. Crowley, president and…
Get regular updates to your inbox.
