Axonal excitability changes in children with spinal muscular atrophy treated with nusinersen

• Published in: The Journal of physiology Vol. 600; no. 1; pp. 95 - 109
• Main Authors: Kariyawasam, Didu S. T., D'Silva, Arlene M., Herbert, Karen, Howells, James, Carey, Kate, Kandula, Tejaswi, Farrar, Michelle A., Lin, Cindy (Shin‐Yi)
• Format: Journal Article
• Language: English
• Published: England Wiley Subscription Services, Inc 01.01.2022
• Subjects: Action potential; Action Potentials; Atrophy; axonal excitability; Axonogenesis; Axons; Child; Child, Preschool; Children; Excitability; Humans; Maturation; Motor neurons; Muscular Atrophy, Spinal - drug therapy; neurobiology; Neurodegeneration; Neurodevelopment; Neuromuscular diseases; nusinersen; Oligonucleotides; SMN protein; Spinal cord; Spinal muscular atrophy; Ventral roots; Wrist; neurobiology; axonal excitability; nusinersen; spinal muscular atrophy; neurodevelopment

Spinal muscular atrophy (SMA) is associated with developmental disruption of motor axons in ventral roots of the spinal cord alongside motor axon degeneration. The pathogenesis of peripheral axonal change during development is pertinent to understand treatment response. Nerve excitability techniques...