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…
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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…
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…
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 feelings. Her personal journey informs the mental health care and advocacy she…
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 with SMA who had previously been treated with gene therapy but did not respond as…
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 new avenues for treating SMA in its earliest stages, researchers say. Zeynep Dokuzluoglu, a graduate…
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 associated with significantly earlier time to first treatment and is subsequently associated with improved…
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, the parent company of this site, sat down with John F. Crowley, president and…
Apitegromab, a muscle-strengthening therapy for spinal muscular atrophy (SMA), helped improve motor function in a new analysis of Phase 3 trial data, with the greatest gains seen in people who started treatment earlier. In the Phase 3 SAPPHIRE study (NCT05156320), participants received apitegromab in addition to disease-modifying…
