Oligonucleotides, Antisense

Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.
DrugDrug NameDrug Description
DB05528MipomersenAn oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.
DB05487CustirsenInvestigated for use/treatment in brain cancer and breast cancer.
DB06759FomivirsenIndicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS), when other therapy has been ineffective or is considered unsuitable [FDA Label, L1428].
DB13811OblimersenNot Annotated
DB06014EteplirsenAn antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) only in patients who are confirmed to have a specific type of mutation.
DB13161NusinersenA medication used to treat spinal muscular atrophy.
DB14713InotersenAn antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
DB15066GivosiranA 5-aminolevulinic acid synthase-directed small interfering RNA (siRNA) used in the prophylaxis of acute hepatic porphyria.
DB15593GolodirsenA drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD).