Twin girls with spinal muscular atrophy (SMA) type 0 are still alive with normal motor function more than two years after treatment with Zolgensma (onasemnogene abeparvovec-xioi). This finding dramatically differs from the typical progression of SMA type 0, the rarest form of the disease, where symptoms…
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Low bone mineral density (BMD), which leaves bones weak and prone to fractures, was found to be prevalent among children with spinal muscular atrophy (SMA) in China in a recent report. Older age, especially beyond a cut-off of 6.3 years, was associated with a higher likelihood of having low…
NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA) — a disease type marked by progressive weakness in the muscles of the throat and mouth, and those closer to the trunk — has been granted orphan drug designation in the European Union. This designation,…
In the absence of treatment, the long-term progression of spinal muscular atrophy (SMA) types 2 and 3 is highly variable and seems to be influenced by factors including a patient’s age and functional status, according to four years of data in a natural history study. Generally, those with better…
Adults with spinal muscular atrophy (SMA) showed alterations in body composition and metabolic disturbances, including changes in blood sugar and fats, that could leave them vulnerable to cardiometabolic complications, a study shows. People with SMA type 2 and those who weren’t ambulatory, meaning they couldn’t walk, showed the…
Newborns diagnosed with spinal muscular atrophy (SMA) who were treated with Evrysdi (risdiplam) before the onset of symptoms are continuing to reach developmental milestones such as sitting, standing, and walking independently, with many achieving them in a time frame expected of typical child development. That’s according to two-year…
A new large-scale genetic screening technique for spinal muscular atrophy (SMA) called comprehensive SMA analysis, or CASMA, was more effective at detecting disease-related mutations in carriers than standard approaches, a study reports. CASMA “is a simple and accurate screening method for SMA, which shows greater clinical efficacy in the…
An experimental high-dose regimen of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) is showing motor benefits for previously untreated patients of all ages, as well as those who switched over from Spinraza’s approved dose, according to new data from the DEVOTE clinical trial. Among babies who’d never received…
Scholar Rock’s apitegromab led to motor function improvements over a year of treatment in young adults and children with spinal muscular atrophy (SMA) type 2 or 3 who could sit independently and were on stable standard of care, but unable to walk. That’s according to…
Treatment for 10 months with Spinraza (nusinersen) normalized the activity of 38 genes in adults with spinal muscular atrophy (SMA), a genetic study showed. Among the normalized genes were those involved in immune signaling pathways — findings that are “in line with the potential of this therapy to…
