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 I’ve seen. Typically, as in “Jurassic World: Rebirth,” genetic editing is framed either as…
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…
Discussion
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 weak, inadequate, and overwhelmed by their compassion. But today is Caregiver Appreciation Day, so…
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,…
Discussion
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 our knowledge and experience to educate others, from friends to medical professionals, about…
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