Study of muscle and pulmonary function of children with SMA, treated with nusinersen

Spinal Muscle Atrophy is a genetic disease that usually presents in childhood. Recently a treatment for the disease, nusinersen, was found to be effective in slowing down or stopping the disease progression. In our study we will be measuring how respiratory function, muscle function and quality of life change over the course of 5 years after treatment start with nusinersen.

Spinal Muscle Atrophy is a genetic disease that usually presents in childhood. Recently a treatment for the disease was found to be effective in slowing down or stopping the disease. In our study we will be measuring how muscle function and breathing function changes over time in children who receive the new treatment.