Development and Clinical Applications of Antisense Oligonucleotide Gapmers. (PubMed, Methods Mol Biol) - Oct 1, 2026 - "Tofersen is an example of a recently FDA-approved antisense oligonucleotide gapmer used for the treatment of amyotrophic lateral sclerosis. Many others are being tested in clinical trials or under preclinical development. This chapter will cover advancements in gapmer treatments for various diseases, including familial hypercholesterolemia, hereditary transthyretin amyloidosis, cancer, familial chylomicronemia syndrome, familial partial lipodystrophy, familial hypertriglyceridemia, Huntington's disease, myotonic dystrophy, prion diseases, and amyotrophic lateral sclerosis."
Journal • Review • Amyloidosis • Amyotrophic Lateral Sclerosis • Cardiac Amyloidosis • CNS Disorders • Dyslipidemia • Familial Chylomicronemia Syndrome • Familial Hypercholesterolemia • Genetic Disorders • Huntington's Disease • Hypertriglyceridemia • Lipodystrophy • Metabolic Disorders • Movement Disorders • Muscular Dystrophy • Myotonic Dystrophy • Oncology
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Faiyza Akil Shaikh; Hidenori Moriyama; Leanna Chan; Pusti Shah; Toshifumi Yokota
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