Molecular Markers In CSF May Help Predict How Well People With Type 2 Or 3 SMA Respond To Nusinersen, Study Finds
July 15, 2025
SMA News Today (7/14, Levinson) reports, “Molecular markers in the liquid that surrounds the brain and spinal cord, called the cerebrospinal fluid (CSF), may help predict how well people with type 2 or 3 spinal muscular atrophy (SMA) respond to Spinraza (nusinersen), a study” found. Investigators “found that certain CSF molecules involved in metabolism, which is the process of breaking down substances to generate energy, differed between those who responded to treatment and those who didn’t.” The “metabolites also varied by SMA type, suggesting distinct biological processes may influence Spinraza’s effectiveness.” The findings were published in Neurological Sciences.