Introduction
FAMILIAL CHYLOMICRONEMIA SYNDROME, DUCHENNE MUSCULAR DYSTROPHY, HEREDITARY TRANSTHYRETIN AMYLOIDOSIS, SPINAL MUSCULAR ATROPHY
Receptor-mediated delivery of an antisense oligonucleotide (ASO) using the ligand-conjugated antisense technology is establishing a new benchmark for antisense therapeutics. The triantennary Nine antisense oligonucleotides (ASOs) have been commercialized to date for indications as diverse as spinal muscular atrophy, hereditary transthyretin amyloidosis, familial chylomicronemia syndrome, and Duchenne muscular dystrophy [Ligand-conjugated antisense technology. All ASOs are conjugated to the same GalNAcSixteen GalNAc