treatment response

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…

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…

NfL protein levels may be helpful biomarker for SMA children: Study

Levels of the neurofilament light protein, known as NfL — which serves as an indicator of neurodegeneration — may be a helpful biomarker for monitoring disease progression and treatment responses in children with spinal muscular atrophy (SMA), a new review study suggests. Across six published studies, culled from more…

Falling levels of 4 proteins in CSF may predict response to Spinraza

A drop in the levels of four proteins in the cerebrospinal fluid (CSF) may be a marker of response to treatment with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), a real-world data study suggests. The predictive value of the four-protein panel — which includes neurofilament light chain,…