Treatment with OAV101 IT, a formulation of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s delivered directly into the spinal canal, safely led to motor improvements for children with spinal muscular atrophy (SMA) type 2 who were at least 2 years old, top-line results from the Phase 3 STEER…
Swallowing function may have improved in spinal muscular atrophy (SMA) patients since disease-modifying therapies (DMT) have become available, a review study suggests. The analysis showed problems with swallowing were prevalent in the years before the emergence of DMTs and that studies after the treatments became available indicated stabilizations…
At Cure SMA’s 13th annual Hope on the Hill event in Washington, D.C., spinal muscular atrophy (SMA) patients, their families, lawmakers, and other advocates gathered to celebrate recent progress in disease research and legislative priorities for the SMA community. Among the speakers at the event were several members…
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…
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…
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…
Babies born prematurely were diagnosed with spinal muscular atrophy (SMA) through newborn screening programs (NBS) in Germany and, in most cases, started on treatment before disease symptoms emerged, a retrospective study reports. Doctors, however, waited to initiate treatment until many of the 12 newborns had reached full-term age,…
Brain abnormalities visible on MRI scans were seven times more likely in people with spinal muscular atrophy (SMA) than in those without the disease, a small study found. Patients with indicators of more severe disease, including having SMA type 1 and fewer copies of the SMN2 gene, had…
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