Spectrum of Neuropathophysiology in Spinal Muscular Atrophy Type I

We present a pathophysiologic model for SMA-I as a protein deficiency disease affecting a neuronal network with variable clinical thresholds. Because new treatment strategies improve survival of infants with SMA-I, a better understanding of these factors will guide future treatments.
Source: Journal of Neuropathology and Experimental Neurology - Category: Neurology Tags: Original Articles Source Type: research