| abstract |
The present disclosure relates compositions and methods to treat neuromuscular diseases and disorders, e.g., spinal muscular atrophy (SMA), characterized by the presence of a splicing silencer located in SMN2 intron 7 pre-mRNA, comprising co-administering a therapeutically effective amount of an antisense oligonucleotide (ASO) complementary to a nucleotide sequence within intron 7 of human SMN2 pre-mRNA; and a subclinical dose of a histone deacetylate inhibitor, e.g., valproic acid, trichostatin A, or a combination thereof. |