An I-STEM team identified moxifloxacin for its ability to increase SMN protein production in a cell-based model screen of already marketed molecules. Administered daily subcutaneously to mouse models of SMN1-related proximal spinal muscular atrophy (SMA), this quinolone antibiotic :
- increases SMN protein production in the spinal cord and skeletal muscle,
- improves the function of neuromuscular junctions and reduces neuroinflammation,
- improves motor performance in mice and extends their life span.
