Get regular updates to your inbox.
Scientists at The Jackson Laboratory — a U.S.-based biomedical research nonprofit — were granted $22.8 million by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), for a novel method to develop gene editing therapies for neurological disorders, including…
Current therapeutic approaches have helped to improve motor function in the majority of children with spinal muscular atrophy (SMA) type 1 in Spain, a study reports. The study, “Clinical and Functional Characteristics of a New Phenotype of SMA Type I among a National Sample of Spanish…
Note: This column describes the author’s own experiences with Evrysdi. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. Surrounded by beeping machines, monitors, wires, and tubes, I had trouble feeling like anything more than a patient during my…
Adults with spinal muscular atrophy (SMA) who start treatment with Spinraza (nusinersen) tend to see the most dramatic early improvement in muscle strength in the shoulders and upper arms, a new study suggests. The treatment also tended to improve thumb strength, which researchers note may be important…
Spinraza (nusinersen) carries relatively few side effects and nearly halves the odds of children and adolescents with spinal muscular atrophy (SMA) having serious or fatal side effects compared with a placebo, according to a meta-analysis. The study, “Adverse events in the treatment of spinal muscular atrophy…
Crying the moment I saw Taylor Swift on stage wasn’t something I expected to happen. But it wasn’t completely unexpected, either. I realize I’m contradicting myself here, but emotions will do that to a person. They’ll feel exhilarating and paralyzing simultaneously, and yet somehow manage to create something cathartic.
The adoption of a newborn screening (NBS) program for spinal muscular atrophy (SMA) in England would reduce healthcare costs and boost the number of healthy years a patient can live, according to a recent cost-effectiveness analysis. Researchers believe such a program could capture most cases of SMA in…
The overactivity of a specific signaling pathway may contribute to the abnormalities in clearing cellular waste that are commonly seen in spinal muscular atrophy (SMA), according to experiments with patient-derived cells. This known cell survival pathway, dubbed the ERK signaling pathway, exhibited increased activity in the cells, while…
The 2002 science-fiction film “Minority Report” imagines a world in which a specialized police unit uses psychic technology to detect crimes before they happen. Tom Cruise plays a detective who’s part of the “Precrime” division, and it’s his job to arrest would-be murderers and criminals. Based on…
I woke up to a text from my friend Sherry: “Babe, you should try this new accessibility tool that just came out. It might help you game!” When it comes to video games, Sherry is the smartest person I know. She freelances as a journalist in the gaming…
Get regular updates to your inbox.