VP226 Post-hoc analysis of compound motor action potential from clinical trials of intravenous onasemnogene abeparvovec for spinal muscular atrophy

Spinal muscular atrophy (SMA) is characterized by muscle weakness, and, therefore, the progression of the disease and the potential efficacy of therapies can be evaluated by means of electrophysiologic recordings. The compound motor action potential (CMAP) summarizes muscle fiber action potentials, and it correlates with the degree of motor nerve innervation, making it potentially valuable as an SMA biomarker. CMAP was evaluated in three completed clinical trials of intravenous onasemnogene abeparvovec for patients with SMA type 1 and two copies of the survival motor neuron 2 gene (SMN2): START (NCT02122952), STR1VE (NCT03306277), and SPR1NT (NCT03505099).
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research