Oligonucleotide therapy has come a long way since the early days. Ongoing research is finding more and more applications for this therapeutic tool. At present there are six FDA approved drugs based on oligonucleotide therapy. These are fomivirsen for treatment of CMV retinitis in AIDS patients, mipomersen for treatment of familial hypercholesterolemia, defibrotide for treatment of venoocclusive disease in liver, eteplirsen for treatment of Duchene Muscular Dystrophy, pegaptanib for treatment of neovascular age related macular degeneration and nusinersen for management of spinal muscular atrophy.
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