Community Perspectives

Up to three years of treatment with Evrysdi (risdiplam) appears safe and was associated with presymptomatic children with spinal muscular atrophy (SMA) reaching and maintaining key developmental milestones, according to Phase 2 study…

A high-dose regimen of Spinraza (nusinersen) that is up for regulatory approval in the U.S. may help stabilize or improve motor function for people with spinal muscular atrophy (SMA), pooled results from two clinical trials…

As a licensed clinical social worker based in Amarillo, Texas, Ali Ramos uses her own experience of living with spinal muscular atrophy (SMA) type 2 to create a safe space for her clients to explore their…

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…

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…

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…

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…

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…

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,…