Antisense oligonucleotides represent a novel therapeutic platform for the discovery of
medicines that have the potential to treat most neurodegenerative diseases. Antisense
drugs are currently in development for the treatment of amyotrophic lateral sclerosis,
Huntington's disease, and Alzheimer's disease, and multiple research programs are
underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has
been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen …