1. Risdiplam: Oral small molecule that promotes SMN2 exon 7 inclusion.2. Branaplam (SMA/Huntington’s): Modulates 5′ SS, discontinued due to safety concerns.3. SF3B1 inhibitors (E7107, Sudemycins, and H3B-8800): Block spliceosome assembly, induce tumor apoptosis.4. PRMT5 inhibitors: Suppress glycolysis and proliferation in bone tumors; regulate DNA repair, enable synthetic lethality with PARP inhibitors.5. SRPK inhibitors: Modulate VEGF splicing to inhibit angiogenesis. The gene discussed is SF3B1; the disease is neoplasm.