SMA treatment

Biogen is very pleased with the “striking” results seen thus far in clinical testing with its therapy candidate salanersen — designed to have high efficacy in people with spinal muscular atrophy (SMA) — and…

Treatment with Evrysdi (risdiplam) is generally safe and effective at improving or stabilizing motor and lung function in adults with spinal muscular atrophy (SMA) for up to two years, according to a real-world study in…

Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA), slowed neurodegeneration, and improved motor function, in children with the genetic disease. The data also demonstrated that…

Injecting medicines into the amniotic fluid — the liquid that surrounds a developing fetus during pregnancy — may be a viable strategy for treating spinal muscular atrophy (SMA) before birth, according to a new…

Richard Finkel, MD, a pediatric neurologist at St. Jude Children’s Research Hospital, was included on a list of 100 individuals having a positive influence on global health in 2025, in recognition of his work treating childhood neuromuscular disorders…

Problems in how dopamine and other chemical messengers work in nerve cells can cause issues with posture in spinal muscular atrophy (SMA), according to a new study in mice that also suggests that levodopa therapy —…

SMA Europe is launching an initiative to share personal stories of how treatment affects the lives of people with spinal muscular atrophy (SMA). The initiative comes as part of OdySMA, an SMA…

Slightly more than half of spinal muscular atrophy (SMA) patients in Australia who gave feedback for a study on how well they thought their treatments worked did not notice any improvement. The researchers said the results suggest a…

Without treatment, most people with spinal muscular atrophy (SMA) who have four copies of the “backup” SMN2 gene will start developing symptoms in early childhood, a study indicates. “The early onset of symptoms in our cohort, coupled with the expected wide clinical variability, strongly supports the characterization of…

A young girl whose muscle weakness and respiratory distress in infancy were treated only as such was diagnosed as a 1-year-old with a severe form of spinal muscular atrophy (SMA), scientists in Pakistan report. She died shortly after her SMA type 1 diagnosis, and after starting on supportive…