During the past seven years, several ASOs have been approved for clinical usage for homozygous familial hypercholesterolemia (Kynamro), spinal muscular atrophy (Spinraza), Duchenne muscular dystrophy (EXONDYS 51), polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis (Tegsedi and Batten disease (Milasen) [32]. The gene discussed is TTR; the disease is juvenile neuronal ceroid lipofuscinosis.