Eteplirsen is an oligonucleotide (roughly speaking, its chemistry resembles that of DNA).
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In order to work out where an oligonucleotide has stuck, an extra, fluorescent, component is added to it when it is made.
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The novel antisense oligonucleotide works by inhibiting the synthesis of apo-B and is under development in the United States and Europe for the treatment of familial hypercholesterolemia.
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