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Zilganersen
go.drugbank.com/drugs/DB18161ApprovedInvestigational[L64096] It is administered by intrathecal injection, and requires dilution with an accompanying artificial cerebrospinal fluid diluent before administration. … [L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons
Vadadustat
go.drugbank.com/drugs/DB12255ApprovedInvestigational[L39610,L46936,L46951] Vadadustat was approved by the FDA in March 2024.[L50371] … One of the most common symptoms of advanced renal disease is anemia, caused primarily by the inability of the kidney to respond to anemic conditions with a corresponding increase in [erythropoietin] (EPO
Xylitol
go.drugbank.com/drugs/DB11195ApprovedInvestigationalXylitol is a non-fermentable sugar alcohol by most plaque bacteria, indicating that it cannot be fermented into cariogenic acid end-products [A32423]. … It works by inhibiting the growth of the microorganisms present in plaque and saliva after it accummulates intracellularly into the microorganism [A32423].
Berberine
go.drugbank.com/drugs/DB04115ApprovedInvestigationalWithdrawnIt is relatively toxic parenterally, but has been used orally for various parasitic and fungal infections and as antidiarrheal.
Lepirudin
go.drugbank.com/drugs/DB00001ApprovedWithdrawn[A3, L41539] HIT is caused by the expression of immunoglobulin G (IgG) antibodies that bind to the complex formed by heparin and platelet factor 4. … Lepirudin is a recombinant hirudin formed by 65 amino acids that acts as a highly specific and direct thrombin inhibitor.
Xamoterol
go.drugbank.com/drugs/DB13781ApprovedIt modulates the sympathetic control of the heart but has no agonist action on β2-adrenoceptors.
Monobenzone
go.drugbank.com/drugs/DB00600ApprovedWithdrawnIt exerts a depigmenting effect on skin of mammals by increasing the excretion of melanin from the melanocytes. It may also cause destruction of melanocytes and permanent depigmentation.
Tivozanib
go.drugbank.com/drugs/DB11800ApprovedInvestigationalIt was approved on March 10, 2021 by the FDA. Marketed by Aveo Oncology, tivozanib is a promising therapy for individuals with RCC who have not been treated successfully with other therapies.[L32524]
Brexanolone
go.drugbank.com/drugs/DB11859ApprovedInvestigationalsusceptible to PPD whereas many commonly used anti-depressive medications elicit actions that may modulate the presence and activity of substances like serotonin, norepinephrine, and/or monoamine oxidase but … As of March 2019, brexanolone - developed and made available commercially by Sage Therapeutics Inc. as the brand name product Zulresso - is the first drug to have ever been approved by the US FDA specifically
Tavaborole
go.drugbank.com/drugs/DB09041ApprovedTavaborole functions by inhibiting Leucyl-tRNA synthetase, or LeuRS, an essential fungal enzyme required for protein synthesis and for the catalysis of ATP-dependent ligation of L-leucine to tRNA(Leu).