The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

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 ...

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 ...

Correspondence to Dr Iain Mayank Dutia, The University of Queensland School of Human Movement and Nutrition Sciences, Saint Lucia, QLD 4072, Australia; i.dutia{at}uq.edu.au Objective This study aims ...