Elevated levels of neutralizing antibodies against AAV9, which can reduce the efficacy of the gene therapy Zolgensma (onasemnogene abeparvovec), were uncommon in infants and young children with spinal muscular atrophy (SMA), a study found. It also showed that anti-AAV9 antibody levels, highest in early infancy, declined over time,…
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Intellectual disability is not common among children with type 2 and 3 spinal muscular atrophy (SMA), but some aspects of cognitive function might be impaired in these children, according to a recent study. In particular, performance in specific tasks involving working memory — easily accessible information retained for a…
A novel smartphone sensor-based assessment tool is able to capture day-to-day changes in motor function in people with spinal muscular atrophy (SMA), according to a proof-of-concept study. The digital tasks are engaging and easy to complete for most SMA patients, and the results are reliable, matching those from established…
A first patient has been dosed in a Phase 2 clinical trial evaluating the safety and efficacy of NMD670, an oral treatment candidate for spinal muscular atrophy (SMA) type 3, its developer, NMD Pharma, announced. Called SYNAPSE-SMA (NCT05794139), the trial is now recruiting adults, ages 18 to…
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 causative…
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 involves…
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 critical…
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 — in…
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 disease-modifying…
