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Betibeglogene autotemcel
go.drugbank.com/drugs/DB16900ApprovedInvestigationalBetibeglogene autotemcel is an autologous gene therapy that adds functional copies of the β-globin gene (β<sup>A-T87Q</sup>-globin) to hematopoietic stem cells in order to treat β-thalassemia. … [L42940] β-thalassemia is a condition caused by mutations in the β-globin gene (HBB) that leads to a significant decrease in the production of β-globin.
Calcium lactate
go.drugbank.com/drugs/DB13231ApprovedVet approvedIt is prepared commercially by the neutralization of lactic acid with calcium carbonate or calcium hydroxide. … Approved by the FDA as a direct food substance affirmed as generally recognized as safe, calcium lactate is used as a firming agent, flavoring agent, leavening agent, stabilizer, and thickener [L2741].
Mixtures: Calcium Magnesium Tablets Nu Life, Pro Enz TabDecitabine
go.drugbank.com/drugs/DB01262ApprovedInvestigational[A2263, A2264, A2265, A215317, L14962] Decitabine was developed by MGI Pharma/SuperGen Inc. and was approved by the FDA for the treatment of MDS on February 5, 2006.
Haloprogin
go.drugbank.com/drugs/DB00793ApprovedWithdrawnThe mechanism of action is unknown, but it is thought to be via inhibition of oxygen uptake and disruption of yeast membrane structure and function. … Haloprogin is no longer available in the United States and has been discontinued.
Grepafloxacin
go.drugbank.com/drugs/DB00365ApprovedWithdrawnDue to the QTc-prolonging potential, as indicated by the changes in the QT interval on the electrocardiogram, and the risk for cardiovascular adverse events, grepafloxacin was withdrawn in the United States
TrenibotulinumtoxinE
go.drugbank.com/drugs/DB19082Approved[L64095] TrenibotulinumtoxinE is distinguished from the serotype A toxins that dominate this setting by its rapid onset and short duration: effects begin around 8 hours after injection, peak at day 7, … [L64095] TrenibotulinumtoxinE was approved by Health Canada in June 2026[L64128] and by the European Commission on July 17, 2026, with the decision applying across all 30 European Economic Area countries
N4-Hydroxycytidine
go.drugbank.com/drugs/DB15660Experimental[A193014] It is orally bioavailable in mice and distributes into tissue before becoming the active 5’-triphosphate form, which is incorporated into the genome of new virions, resulting in the accumulation … [A193011] In non-human primates, N4-hydroxycytidine was poorly orally bioavailable.
Synonyms: 4-N-Hydroxycytidine, N(4)-HydroxycytidineLonafarnib
go.drugbank.com/drugs/DB06448ApprovedInvestigationalaberrant farnesylated form of lamin A called progerin in the inner nuclear membrane. … [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
NV-07a
go.drugbank.com/drugs/DB05845ExperimentalNV-07a is topical skin repair compound protecting skin from sun-induced immunosuppression and UV-induced damage.
Resmetirom
go.drugbank.com/drugs/DB12914ApprovedInvestigationalOn March 14, 2024, it was approved by the FDA as the first treatment of liver fibrosis due to noncirrhotic non-alcoholic steatohepatitis (NASH), which is a form of non-alcoholic fatty liver disease (NAFLD … [A263461] Resmetirom works to reduce liver fat by stimulating fatty acid degradation and oxidation.[L50336]