Motor unit changes in children with symptomatic spinal muscular atrophy treated with nusinersen.
Didu S T KariyawasamArlene D'SilvaJames HowellsKaren HerbertPeter Geelan-SmallCindy Shin-Yi LinMichelle Anne FarrarPublished in: Journal of neurology, neurosurgery, and psychiatry (2020)
Nusinersen therapy facilitated functional innervation in SMA through recovery of smaller motor units. Delineation of biomechanisms of therapeutic response may be the first step in identifying potential novel targets for disease modification in this and other motor neuropathies. MScanFit MUNE techniques may have a broader role in establishing biomarkers of therapeutic response in similar adult-onset diseases.