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…
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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…
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…
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…
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…
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.
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.
Biogen has launched a trio of trials to test its new drug candidate for spinal muscular atrophy (SMA), salanersen, which aims to be a more convenient successor to the company’s approved SMA therapy…
Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research…