Figure 4.

Landscape of targets, drugs, therapeutic strategies, and treatments for SMA. Therapeutic strategies have focused on SMN-dependent approaches, such as the modification of SMN2 splicing through antisense oligonucleotides and small molecules, the increment of SMN transcripts through histone deacetylase (HDAC) inhibitors, and replacement of defective SMN1 through gene therapy, and SMN-independent approaches, such as neuroprotection, cell therapy through stem cell transplantation, muscle enhancement through myostatin inhibitors and fast troponin activators, and the improved function of neuromuscular junction.