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Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a…
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,…
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…
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…
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…
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…
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