Furthermore, our molecular investigation confirmed a direct link between miRNA-206-HDAC4 and FGFBP1 in vitro and in vivo, and that this pathway is up-regulated in a late phase of the disease, suggesting that miRNA-206 could be an innovative, still relatively unexplored, strategy to treat SMA. The gene discussed is FGFBP1; the disease is proximal spinal muscular atrophy.