Approaches such as antisense oligonucleotides (ASOs) and small molecules that modulate splicing have shown promise in treating other genetic disorders, such as spinal muscular atrophy (SMA).[55, 56, 57] Similar strategies may be explored to correct the splicing defects in CASPASE3 and AIFM1 caused by EFTUD2 mutations in patients. The gene discussed is EFTUD2; the disease is proximal spinal muscular atrophy.