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Lonafarnib
go.drugbank.com/drugs/DB06448ApprovedInvestigational[A224379, A224384, A224389, A224394, A224399] Mechanistically, HGPS is underpinned by a single heterozygous C-to-T mutation at position 1824 of the _LMNA_ gene, which results in the accumulation of an … for localization to the nuclear membrane, lonafarnib inhibits progerin accumulation and improves symptoms in HGPS patients.
Categories: Alimentary Tract and Metabolism, Various Alimentary Tract and Metabolism ProductsDeutetrabenazine
go.drugbank.com/drugs/DB12161ApprovedInvestigationalThe most prominent physical symptom of HD that may increase the risk of injury is chorea, which is an involuntary, sudden movement that can affect any muscle and flow randomly across body regions [A32046 … It was approved by FDA in April 2017 and is marketed under the trade name Austedo as oral tablets.
Categories: Tetrabenazine and isomerVorasidenib
go.drugbank.com/drugs/DB17097ApprovedInvestigational[A264209] It works by suppressing the levels of D-2-hydroxyglutarate (2-HG), an oncometabolite produced by mutant IDH1 and IDH2 isoforms. … Vorasidenib is a first-in-class dual isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor.
Categories: Antineoplastic and Immunomodulating AgentsMethoxyflurane
go.drugbank.com/drugs/DB01028ApprovedInvestigationalVet approvedWithdrawnAn inhalation anesthetic. Currently, methoxyflurane is rarely used for surgical, obstetric, or dental anesthesia. … (From AMA Drug Evaluations Annual, 1994, p180) In the US, methoxyflurane is one of the products that have been withdrawn or removed from the market for reasons of safety or effectiveness.[L43942]
Categories: Miscellaneous Analgesics and AntipyreticsLevocabastine
go.drugbank.com/drugs/DB01106ApprovedLevocabastine is a selective second-generation H1-receptor antagonist used for allergic conjunctivitis. Levocabastine was discovered at Janssen Pharmaceutica in 1979.
Categories: Decongestants and AntiallergicsLevobetaxolol
go.drugbank.com/drugs/DB09351ApprovedIt was marketed as a 0.5% ophthalmic solution of levobetaxolol hydrochloride under the trade name Betaxon but has been discontinued. … Levobetaxolol is a beta-blocker used to lower the pressure in the eye to treat conditions such as glaucoma.
Avacincaptad pegol
go.drugbank.com/drugs/DB15165ApprovedInvestigationalIt was developed to treat an advanced form of age-related macular degeneration (AMD) called geographic atrophy (GA). … Avacincaptad pegol is an RNA aptamer covalently bound to a branched polyethylene glycol (PEG) molecule.
Categories: Nucleic Acids, Nucleotides, and NucleosidesAvatrombopag
go.drugbank.com/drugs/DB11995ApprovedInvestigationalThis approval was supported by results from the AVA-PED-301 phase 3 study, which demonstrated durable platelet responses and favorable safety in children with persistent or chronic immune thrombocytopenia … [F95] Avatrombopag was first approved by the FDA in May 2018 for use in adults with chronic liver disease who are scheduled to undergo a procedure.
Categories: Blood and Blood Forming OrgansEnmetazobactam
go.drugbank.com/drugs/DB18716ApprovedInvestigational[A263266] Because ESBL enzymes can hydrolyze important antibiotics such as penicillins, broad-spectrum cephalosporins and monobactams, ESBL-producing bacteria poses challenges in the treatment of serious … [A263276] The combination product of enmetazobactam and [cefepime] was first approved by the FDA on February 23, 2024, for the treatment of complicated urinary tract infections.