Genes, Volume 15, Issue 7 (July 2024) – 146 articles
Cover Story (view full-size image):
Historically, the development of therapies used to treat rare diseases has been heavily restricted by low patient numbers and high development costs that impede traditional clinical trials. However, several recent N-of-1 trials using small DNA-like molecules known as antisense oligonucleotides have highlighted the immense potential that these molecules may have as therapeutics for rare diseases. In this review, the authors outline the development of N-of-1 antisense therapies to date, including the landmark case of Milasen, as well as provide an overview of the changing FDA regulations surrounding antisense therapies for rare disease indications. View this paper
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