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A targeted test looking for mutations in known disease-associated genes in spinal muscular atrophy (SMA) was found to be a useful first step in identifying the cause of rare types of SMA, a new study reports. In fact, according to the researchers, “a total of ten patients with…
Researchers in Turkey have developed a new method for screening newborn blood samples for mutations associated with spinal muscular atrophy (SMA). Compared with standard techniques, the new method showed 100% accuracy for detecting SMA-positive and negative samples, they said. The scientists believe their approach, which is simpler and…
Scholar Rock announced that a Phase 3 clinical trial of its experimental muscle-targeting therapy apitegromab has finished enrolling children and young adults with spinal muscular atrophy (SMA) types 2 or 3, who are unable to walk but can sit independently. “Completing SAPPHIRE enrollment is a…
The Muscular Dystrophy Association (MDA) has awarded one of 37 new research grants to a professor studying the role of survival motor neuron or SMN — the protein that’s missing in people with spinal muscular atrophy (SMA) — in disease development and treatment. Winning the three-year grant —…
Biohaven Pharmaceuticals has completed the enrollment of children and young adults with spinal muscular atrophy (SMA) in a pivotal Phase 3 trial in the U.S. and Europe that is testing its experimental muscle-targeting therapy taldefgrobep alfa. The company had expected to recruit about 180 patients of all…
Children with spinal muscular atrophy (SMA) treated with Zolgensma (onasemnogene abeparvovec) used fewer healthcare resources and had lower medical costs than children on Spinraza (nusinersen), but they had higher SMA treatment costs, according to a recent U.S. analysis. Overall, healthcare costs for SMA patients using these…
Adults with spinal muscular atrophy (SMA) experience more health problems affecting a range of bodily systems compared with the general population, according to a U.S. healthcare claims analysis. A total of 30 conditions examined in the study, many of which are associated with older age in the general…
Spinraza (nusinersen) led to clinically meaningful motor improvements for three of six adults with spinal muscular atrophy (SMA) types 2 or 3 who were treated at a hospital in Japan. Other aspects, including respiratory and swallowing function, did not change considerably. “The distinct efficacy of [Spinraza] requires…
Evrysdi (risdiplam) safely stabilizes or improves motor function for most people with spinal muscular atrophy (SMA) types 1, 2, or 3, according to a new meta-analysis that reviewed more than a half-dozen studies of the approved therapy. The findings related to respiratory function were less consistent, however,…
The European Commission (EC) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old, making the therapy now available to treat SMA patients across all age ranges in Europe. Evrysdi was first approved in the European Union…
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