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…
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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…
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 Spinraza (nusinersen). Salanersen would need to be administered just once per year. The biotech…
Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in adults with spinal muscular atrophy (SMA), and showed a manageable safety profile, according to a nationwide study in South Korea. Among 19 adults, researchers reported meaningful gains in motor function scores — particularly in knee bending and…
Scholar Rock plans to resubmit its application to the U.S. Food and Drug Administration (FDA) seeking approval for apitegromab, an investigational muscle-strengthening therapy for spinal muscular atrophy (SMA). The submission is contingent on the successful FDA reinspection of a manufacturing site operated by the company’s third-party fill-finish…
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 and care. Ahead of the event, Bionews, the parent company of this site, sat…
In children with spinal muscular atrophy (SMA) type 1, the most severe form of the genetic disease, treatment with Spinraza (nusinersen) restored levels of taurine — an amino acid that plays a role in the delivery of information between nerve cells — which were found to be low,…
The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular atrophy (SMA) type 1. “I would like to express our gratitude for the young child and family who were courageous in being the first to accept our investigational gene therapy…
