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…
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…
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…
To its credit, “The Beauty,” a new sci-fi, body-horror miniseries from Ryan Murphy, co-creator of “American Horror Story” and other acclaimed projects, dives deeper into the topic of genetic editing and disability than most sci-fi shows…
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…
How can I possibly show the deep appreciation that I have for the caregivers in my life with spinal muscular atrophy (SMA)? This is a question that burdens me often when I feel…
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…
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…
When I was little, SMA wasn’t widely known or understood. Its rarity meant that few people had heard of it unless they were confronted with it. Families like mine carried much of the responsibility of sharing…
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…
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