A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data from the SMArtCARE registry. Researchers not only observed a stabilization of disease progression or lack of deterioration, but clinically meaningful improvements in walking distance in a subgroup of patients. The findings of the study have been published in the Journal of Neuromuscular Diseases.
SMArtCARE is a disease-specific registry with 58 participating centers in Germany, Austria and Switzerland. Data are collected as real-world data during routine patient visits. The study’s analysis included all patients being treated with nusinersen able to walk independently before the start of treatment, with a focus on changes in motor function.
