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Idebenone
go.drugbank.com/drugs/DB09081ApprovedInvestigationalLack of improvement in cognitive function halted its production for these conditions, however it continues to be investigated for use in other conditions associated with mitochondrial damage. … LHON is a mitochondrially inherited degeneration of retinal ganglion cells, resulting in acute central vision loss.
Categories: Compounds used in a research, industrial, or household settingVanzacaftor
go.drugbank.com/drugs/DB18373ApprovedInvestigational[L52275,A179677] Vanzacaftor was first approved by the US FDA in December 2024 in combination with another CFTR corrector - [tezacaftor], which binds to a different site than vanzacaftor - and [deutivacaftor … It is used alongside other CFTR correctors and CFTR potentiators to increase the quantity and function of CFTR at the cell surface in patients with cystic fibrosis.
Enprofylline
go.drugbank.com/drugs/DB00824ApprovedWithdrawnEnprofylline is used in asthma, chronic obstructive pulmonary disease, and in the management of cerebrovascular insufficiency, sickle cell disease, and diabetic neuropathy. … Long-term enprofylline administration may be associated with elevation in liver enzyme levels and unpredictable blood levels.
Synonyms: 3-n-propylxanthineIptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigational[A262581] On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. … This approval was based on favorable results obtained from the phase III APPL-PNH and APPOINT-PNH studies, where 82.3% and 77.5% of patients experienced a sustained hemoglobin improvement without transfusions
Categories: Compounds used in a research, industrial, or household settingZolmitriptan
go.drugbank.com/drugs/DB00315ApprovedInvestigational[A193791] Triptans can be administered alone or in combination with an NSAID like [naproxen], and represent the current "gold standard" for acute migraine treatment. … It is currently available in both tablet and nasal spray forms.[L12978]
Acemetacin
go.drugbank.com/drugs/DB13783ApprovedInvestigational[A31352] In clinical trials, acemetacin exhibits a better gastric tolerability compared to its active metabolite indometacin.[T50] It was developed by E. … Merck and Company in Germany as an attempt to provide a safer drug but other than the amelioration on the gastrointestinal effects, the metabolism of acetamicin led to the formation of indomethacin and
Pariglasgene brecaparvovec
go.drugbank.com/drugs/DB16801Approvedcontinued approval potentially contingent on confirmatory trials. … [L64050] Restoring hepatic G6Pase activity supports the release of glucose during fasting, and in clinical studies reduced patients' reliance on dietary cornstarch used to prevent hypoglycemia.
Mafenide
go.drugbank.com/drugs/DB06795ApprovedVet approvedWithdrawnIn November 2022, the use of mafenide acetate (powder for 5% topical solution) was withdrawn by the FDA due to an unresolved confirmatory study required to fulfill the accelerated approval requirements … [L36773] In 1998, mafenide acetate was approved under the FDA’s accelerated approval regulations.
Fexinidazole
go.drugbank.com/drugs/DB12265Approved[A237555, A237560] Fexinidazole received a positive opinion from the European Medicines Agency (EMA) in November 2018 and was approved by the FDA on July 16, 2021. … Transmitted by the bite of an infected tsetse fly, HAT is biphasic with a first (hemolymphatic) stage that progresses to a second (meningoencephalitic) stage in which patients experience progressively
Tagraxofusp
go.drugbank.com/drugs/DB14731ApprovedInvestigationala myeloid malignancy in the dendritic cell lineage. … [A253762] It was also approved by the European Commission on January 7, 2021.[L43712]