Nusinersen is an antisense oligonucleotide (ASO) specifically designed to treat Spinal Muscular Atrophy (SMA), a severe genetic disorder characterized by the loss of motor neurons, leading to progressive muscle weakness and atrophy. SMA is one of the most common genetic causes of infant mortality. Nusinersen works by modifying the splicing of the SMN2 gene to increase the production of functional survival motor neuron (SMN) protein.
