Spinraza

Inflammatory signaling molecules may predict response to Spinraza

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…

Spinraza aids motor function for SMA children at center in China

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…

Spinraza treatment improves motor function in nonambulatory SMA

Treatment with Spinraza (nusinersen) improved motor function in children, adolescents, and adults with spinal muscle atrophy (SMA) who were unable to walk without assistance, according to a four-year real-world analysis. Moreover, nine of the 12 patients who underwent continuous evaluations while on Spinraza over the four years…

New lab test may help in studying effects of Spinraza treatment

Researchers have developed a new laboratory technique for measuring levels of Spinraza (nusinersen), an approved treatment for spinal muscular atrophy (SMA), in biological fluids. The test is expected to help overcome some limitations of other approaches now in use, and may therefore aid scientists and physicians when studying the…

MDA 2024: Spinraza may benefit some SMA children after Zolgensma

In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), subsequent treatment with Spinraza (nusinersen) can improve motor function and may reduce nerve damage. That’s according to interim data from the Phase 4 RESPOND study…

Long-term Spinraza found safe, effective across SMA spectrum

Long-term treatment with Spinraza (nusinersen) was associated with improved or stable motor function in patients across the spinal muscular atrophy (SMA) disease spectrum, according to an analysis of real-world registry data. “To date, this is the largest prospective study over the longest observational period of [Spinraza] therapy…

Proper nutrition can be lacking in children on Spinraza, study finds

  Difficulties with adequate nutrition and feeding persist among children and adolescents with spinal muscular atrophy (SMA) types 2 and 3 being treated with Spinraza (nusinersen), a Norwegian study reports. Important nutrients, such as protein, fiber, vitamins, and minerals, often were consumed in lower-than-recommended amounts by these patients,…