ASO	Antisense Oligonucleotide
CMC	Chemistry, Manufacturing, and Controls
CTiD	Clinical-Trials-in-a-Dish
DMD	Duchenne Muscular Dystrophy
EU	European Union
hATTR	Hereditary Transthyretin-Mediated Amyloidosis
IND	Investigational New Drug
iPSC	Induced Pluripotent Stem Cell
NAM	New Approach Methodology
PK/PD	Pharmacokinetics/Pharmacodynamics
siRNA	Small Interfering RNA
SMA	Spinal Muscular Atrophy
U.S. FDA	United States Food and Drug Administration
WHO	World Health Organization