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Research and Development of Oligonucleotide Therapeutics in Japan for Rare Diseases

2022-02-28

Abstract excerpt

Inherited gene mutations, insertions, deletions of single genes cause most of the rare diseases. Oligonucleotide therapeutics represent one of the most flexible platforms for developing drugs for rare diseases. Presently, 15 oligonucleotide therapeutics have been approved in the United States of America (USA) to treat various rare diseases and 4 oligonucleotide therapeutics (eteplirsen, golodirsen, viltolarsen and...

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Identifiers and source

Literature Corpus work
393526ed-a261-5abe-be00-4912fb1b658c
DOI
10.2217/frd-2021-0008
Open publication

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