Evrysdi improved motor function and quality of life in children and adults with SMA, a real-world study in Hong Kong shows.
Analyzing data from a phase 2 study of patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA,) researchers found that the assessment is a valid, reliable, and responsive assessment ...
Electrical signals called far-field potentials may help track disease progression in SMA, a new study suggests.