Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may stabilize respiratory muscle strength and delay [noninvasive ventilation] dependence, despite progressive restrictive lung disease,” the…

First-line Zolgensma (onasemnogene abeparvovec-xioi) outperformed first-line Spinraza (nusinersen) in achieving a more sustained clinical response and reducing the need for supportive care among young children with spinal muscular atrophy (SMA) type 1, according to a head-to-head analysis. Still, both therapies led to comparable, clinically meaningful improvements in…

It’s a running joke in my family that, if we had the money, we’d build me a robot caregiver like Baymax from Disney’s “Big Hero 6.” Training human caregivers is nothing short of exhausting. Humans need food and sleep, they forget things, and most problematic of all, they have their…

While I understand that inanimate objects cannot have a mind of their own, there’s a lamp in my bedroom that would kindly disagree. I’ve had this lamp for a couple of years now. It’s set to a timer that turns on every night at 11 p.m. and stays lit until…

Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened bones, according to a study done in mice and cells. The findings suggest that treatments aiming to normalize bone cell communication could help improve bone health in SMA patients, researchers…