In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results show. These findings come from the STEER Phase 3 trial (NCT05089656), which supported…
Discussion
A stool. A chain. A person’s body. None of these things is what I’d usually call “art materials,” but that’s exactly how contemporary Singaporean artist Amanda Heng sees them. During a recent trip to the National Gallery Singapore, I discovered “Walking The Stool,” a 1999 performance by…
Discussion
It is always an interesting and fun challenge to navigate my limits and needs while living on my own with SMA. I have a wonderful community of friends and caregivers who come to my house throughout the day and night to assist me. Still, I am constantly on the…
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 results. “The majority of children treated with [Evrysdi] before the onset of SMA symptoms, maintained…
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 show. “Overall, the improvements observed exceeded what would be expected in a broad…
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