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Displaying all 18 drugs

Viltolarsen

Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive ambulatory, pulmonary, and cardiac function and is invariably fatal. A related, albeit a less severe, form of muscular dystrophy known as Becker muscular dystrophy (BMD) is characterized by...
Approved
Investigational
Matched Synonyms: … Exon 53 specific antisense morpholino oligonucleotide …
Matched Description: … [A218171, A218176, A218191] The application of antisense oligonucleotides in DMD patients with specific …
Matched Categories: … Antisense Oligonucleotides …

Tofersen

Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.[A259028,A259033] Tofersen was granted accelerated approval from the FDA...
Approved
Investigational
Matched Synonyms: … Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 Gene …
Matched Description: … Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 …
Matched Categories: … Antisense Oligonucleotides …

Golodirsen

Golodirsen is a morpholino antisense oligomer designed to treat about 8% of patients with Duchenne Muscular Dystrophy (DMD). This is an X-linked condition leading to progressive muscle degeneration that begins in early childhood, rendering many patients wheelchair-bound by age 12. Often, patients succumb to this condition by age 30 or...
Approved
Matched Description: … Golodirsen is a morpholino antisense oligomer designed to treat about 8% of patients with Duchenne Muscular …
Matched Categories: … Antisense Oligonucleotides ... Antisense Elements (Genetics) …

Givosiran

Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, a critical enzyme in the heme biosynthesis pathway. It is manufactured by Alnylam Pharmaceuticals and was first approved for use in the United States in November 2019 for the treatment of adults with acute hepatic porphyria, a genetic...
Approved
Investigational
Matched Categories: … RNA, Antisense ... Antisense Oligonucleotides …

Inotersen

Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults. It was FDA approved in October 2018. Inotersen has been shown to improve the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis . Hereditary transthyretin...
Approved
Investigational
Matched Description: … Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy …
Matched Categories: … DNA, Antisense ... Antisense Oligonucleotides ... Antisense Elements (Genetics) ... Oligodeoxyribonucleotides, Antisense …

Mipomersen

Mipomersen sodium, which was known as the investigational drug, isis-301012, is the salt form of a synthetic phosphorothioate oligonucleotide. Mipomersen sodium prevents the formation of apo B-100, resulting in a decrease in the levels of apolipoprotein B (apo B), low density lipoprotein (LDL), and total cholesterol. Mipomersen is indicated in...
Approved
Investigational
Matched Categories: … DNA, Antisense ... Antisense Oligonucleotides ... Antisense Elements (Genetics) ... Oligodeoxyribonucleotides, Antisense …

Nedosiran

Nedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate. Oxalate, particularly calcium oxalate, precipitation is the main cause of kidney stones formation; therefore, blocking the production of oxalate can help alleviate renal symptoms. Nedosiran was approved by the FDA on...
Approved
Investigational

Fomivirsen

Fomivirsen is a antisense 21 mer phosphorothioate oligonucleotide. It is an antiviral agent that was used in the treatment of cytomegalovirus retinitis (CMV) in immunocompromised patients, including those with AIDS. As a complementary nucleotide to the messenger RNA of the major immediate-early region proteins of human cytomegalovirus, it disrupts the...
Approved
Investigational
Withdrawn
Matched Description: … Fomivirsen is a antisense 21 mer phosphorothioate oligonucleotide. ... Fomivirsen is marketed under the trade name Vitravene for intravitreal injection and was the first antisense ... immediate-early region proteins of human cytomegalovirus, it disrupts the replication of the virus through an antisense …
Matched Categories: … Antisense Oligonucleotides ... Antisense Elements (Genetics) …

Patisiran

Parisiran is a first in class short interfering RNA for the treatment of patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis . It is marketed as Onpattro which is formulated as patisiran within a liposome envelope for better delivery to the liver, where transthyretin is produced. The approval for Onpattro...
Approved
Investigational
Matched Categories: … RNA, Antisense ... Antisense Elements (Genetics) …

Nusinersen

An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved by the U.S. FDA in December, 2016 as Spinraza for the treatment of children and adults with spinal muscular atrophy (SMA). It is adminstrated as direct intrathecal injection.
Approved
Investigational
Matched Description: … An antisense oligonucleotide that induces survival motor neuron (SMN) protein expression, it was approved …
Matched Categories: … Antisense Oligonucleotides …

Inclisiran

Inclisiran is a long-acting, synthetic small interfering RNA (siRNA) directed against proprotein convertase subtilisin-kexin type 9 (PCSK9), which is a serine protease that regulates plasma low-density lipoprotein cholesterol (LDL-C) levels. By binding to PCSK9 messenger RNA, inclisiran prevents protein translation of PCSK9, leading to decreased concentrations of PCSK9 and plasma...
Approved
Investigational
Matched Categories: … RNA, Antisense ... Antisense Elements (Genetics) …

Casimersen

Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive impairment of ambulatory, pulmonary, and cardiac function and is invariably fatal. A related, albeit a less severe, form of muscular dystrophy known as Becker muscular dystrophy (BMD) is...
Approved
Investigational
Matched Description: … [A218171, A218176, A229988] The application of antisense oligonucleotides in DMD patients with specific …
Matched Categories: … Antisense Oligonucleotides …

Trioxsalen

Trioxsalen (trimethylpsoralen, trioxysalen or trisoralen) is a furanocoumarin and a psoralen derivative obtained from several plants, mainly Psoralea corylifolia. Like other psoralens it causes photosensitization of the skin. It is administered either topically or orally in conjunction with UV-A (the least damaging form of ultraviolet light) for phototherapy treatment of...
Approved
Matched Description: … and used to visualize sites of DNA damage.[3] The compound is has been explored for development of antisense …

Vutrisiran

Vutrisiran is a double-stranded small interfering ribonucleic acid (siRNA) that targets wild-type and mutant transthyretin (TTR) messenger RNA (mRNA). This siRNA therapeutic is indicated for the treatment of neuropathies associated with hereditary transthyretin-mediated amyloidosis (ATTR), a condition caused by mutations in the TTR gene. More than 130 TTR mutations have...
Approved
Investigational
Matched Categories: … RNA, Antisense ... Antisense Oligonucleotides …

Volanesorsen

Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA to prevent its translation. It is indicated to treat familial chylomicronemia, a genetic condition that prevents breakdown of triglycerides and chylomicrons. This drug is not commonly prescribed as it is used as an adjunct to diet in patients at high...
Approved
Investigational
Matched Description: … Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA to prevent its translation. …

Lumasiran

Lumasiran is a small interfering RNA used in the treatment of primary hyperoxaluria type 1 (PH1). This condition, caused by a deficiency in the enzyme alanine-glyoxylate aminotransferase, leads to an accumulation of oxalate, causing calcium crystal formation. These patients experience frequent kidney stones, nephrocalcinosis, and renal failure. Oxlumo, producted by...
Approved
Investigational
Matched Categories: … RNA, Antisense ... Antisense Elements (Genetics) …

Eteplirsen

Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer. It consists of a six-membered morpholino ring replacing the five-membered ribofuranosyl rings found in natural DNA and RNA. Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory...
Approved
Investigational
Matched Description: … Eteplirsen is a synthetic antisense oligonucleotide and a phosphorodiamidate morpholino oligomer. …
Matched Categories: … Antisense Oligonucleotides …

Eplontersen

Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery. It was previously investigated as a potential treatment for hereditary transthyretin-mediated amyloidosis. Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of...
Approved
Investigational
Matched Description: … Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl …
Displaying all 18 drugs