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Some 70% of children with spinal muscle atrophy (SMA) type 1 who underwent treatment with Spinraza alone or with Spinraza and then Zolgensma reached some motor milestones over 18 months, with greater improvements in those who received Spinraza…
Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with spinal muscular atrophy (SMA), a study reported. The study, “Performance fatigability in adults with spinal muscular…
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…
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 —…
The use of ultrasound to guide the administration of Spinraza (nusinersen) injection into the spinal canals of adults with spinal muscle atrophy (SMA) significantly reduced the number of needle redirections compared with a standard, landmark-based approach, according to a clinical trial. Among patients with spinal deformities, ultrasound…
Treatment with an investigational high-dose regimen of Spinraza (nusinersen) was well tolerated and significantly improved motor function in infants with spinal muscular atrophy (SMA) relative to untreated patients, according to top-line data from the pivotal portion of a Phase 2/3 clinical trial. Results from the DEVOTE…
Most children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) in a real-world setting had normal lab test results on measures of treatment safety, according to a new analysis. “These data should provide useful information for clinicians to assess the safety of [Spinraza] therapy in the…
In people with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), levels of pro-inflammatory signaling molecules tend to decrease while levels of anti-inflammatory signaling molecules tend to increase in the months after starting treatment, a study has found. Findings also suggested that changes in certain inflammation-regulating molecules…
Treatment with Spinraza (nusinersen) led to improvements in motor function for nearly all children with spinal muscular atrophy (SMA) who received the therapy at a center in China, and among children who didn’t experience improvements, motor function was generally stable with Spinraza. The findings were detailed in a…
People with spinal muscular atrophy (SMA) who are treated with Spinraza (nusinersen) are less likely to require hospitalization or emergency medical care, and hospitalization-related costs correspondingly tend to decrease after starting on the therapy, a U.S. database study reports. The study, “Evaluation of inpatient and…
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