Through the use of patient testimonials as well as analysis of data from a study of patients with spinal muscular atrophy (SMA), researchers were able to describe patient-centered “meaningful outcomes ...

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately measure ...

When determining the effects of a pharmacological treatment on motor capabilities, a proper tool should be employed. Standard motor tests, including open field or rotarod, are often insufficient at ...