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Deutivacaftor
go.drugbank.com/drugs/DB15141ApprovedInvestigationalIt is used alongside CFTR correctors to increase the quantity and function of CFTR at the cell surface in patients with cystic fibrosis. … [L52275,A179677] Deutivacaftor was first approved by the US FDA in December 2024 in combination with two CFTR correctors - [tezacaftor] and [vanzacaftor] - for the treatment of patients with responsive
Ruxolitinib
go.drugbank.com/drugs/DB08877ApprovedInvestigational[A229708] In 2014, it was approved for the treatment of polycythemia vera in adults who have an inadequate response to or are intolerant of [hydroxyurea] and in 2019, ruxolitinib was approved for use in … In phase II clinical trials, ruxolitinib improved chest computed tomography and improved recovery in patients with lymphopenia.
Elasomeran
go.drugbank.com/drugs/DB15654ApprovedInvestigational[L28026] A phase I, open-label, dose-ranging clinical trial (NCT04283461) was initiated in March 2020 in which 45 subjects received two intramuscular doses (on days 1 and 29). … In clinical trials, there were no differences in the safety profiles between younger and older (65 years of age and older) study participants.
Choline C 11
go.drugbank.com/drugs/DB09277Approved[A32041] It was developed by MCPRF and FDA first approved in September 2012. … Choline C 11 is a marker for cellular proliferation as its main molecule is a precursor for the biosynthesis of phospholipids which are essential components of all cell membranes.
Sonidegib
go.drugbank.com/drugs/DB09143ApprovedInvestigationalSonidegib is a Hedgehog signaling pathway inhibitor (via smoothened antagonism) developed as an anticancer agent by Novartis. It was FDA approved in 2015 for the treatment of basal cell carcinoma.
Synonyms: N-[6-(cis-2,6-dimethylmorpholin-4-yl)pyridin-3-yl]-2-methyl-4'-(trifluoromethoxy)[1,1'-biphenyl]-3-carboxamideOxybutynin
go.drugbank.com/drugs/DB01062ApprovedInvestigationalefficacy since initial FDA approval in 1975. … It is often used as first-line therapy for OAB.[A185990]
Praziquantel
go.drugbank.com/drugs/DB01058ApprovedInvestigationalVet approved[A263201] The efficacy of praziquantel in treating parasitic flatworms infection with low cost (~US$0.20 drug cost to treat a child) makes it an integral to WHO's plan to eliminate schistosomiasis by 2030 … Specifically, it is known as a treatment of trematodes and cestodes infections such as schistosomiasis, taeniasis, and cysticercosis.
Setmelanotide
go.drugbank.com/drugs/DB11700ApprovedInvestigational[A224449] Earlier attempts at agonizing MC4R (such as LY2112688) lead to successful weight loss, but also an increase in blood pressure and heart rate. … [L24474] It is an agonist of the melanocortin 4 receptor.
Acoramidis
go.drugbank.com/drugs/DB17999ApprovedInvestigationalAcoramidis is a small molecule stabilizer of transthyretin (TTR) for use in patients with TTR amyloidosis. … [A264808] It was brought to market by BridgeBio Pharma and approved by the FDA in November 2024 to reduce negative cardiovascular outcomes in patients with cardiomyopathy caused by TTR amyloidosis.
Escitalopram
go.drugbank.com/drugs/DB01175ApprovedInvestigational[A185420] It is used to restore serotonergic function in the treatment of depression and anxiety. … escitalopram exerts the highest degree of selectivity for the serotonin transporter (SERT) relative to other off-targets which may explain its lower rates of adverse effects as compared to other agents in
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