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Raloxifene
go.drugbank.com/drugs/DB00481ApprovedInvestigationalDue to the decline in estrogen levels in postmenopausal osteoporosis, hormone replacement therapy (HRT), such as estradiol, has been used to ameliorate the condition. … However, due to the off-target actions by HRT, newer non-hormonal agents such as raloxifene and [tamoxifen] have been developed to reduce adverse events through selective pharmacological actions on tissue-specific
Palovarotene
go.drugbank.com/drugs/DB05467Approved[A244920] FOP is caused by a gain-of-function mutation in the ACVR1/ALK2 gene which results in progressive heterotopic ossification, a process wherein connective tissues (e.g. skeletal muscle, ligaments … Treatment options for patients with FOP are extremely limited, although there has been substantial recent interest in novel treatments for this disease.
Lovastatin
go.drugbank.com/drugs/DB00227ApprovedInvestigational[A181087,A181553] Use of statins to target and reduce LDL levels has been shown in a number of landmark studies to significantly reduce the risk of development of CVD and all-cause mortality. … rosuvastatin] is considered the most potent; doses of 10 to 40mg [rosuvastatin] per day were found in clinical studies to result in a 45.8% to 54.6% decrease in LDL cholesterol levels, while lovastatin has been
Golodirsen
go.drugbank.com/drugs/DB15593ApprovedInvestigationalGolodirsen is a morpholino antisense oligomer designed to treat about 8% of patients with Duchenne Muscular Dystrophy (DMD). This is an X-linked condition leading to progressive muscle degeneration that begins in early childhood, renderi...
Enasidenib
go.drugbank.com/drugs/DB13874ApprovedInvestigationalEnasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2) gene … This small molecule acts as an allosteric inhibitor of mutant IDH2 enzyme to prevent cell growth, and it also has shown to block several other enzymes that play a role in abnormal cell differentiation.
Valoctocogene roxaparvovec
go.drugbank.com/drugs/DB15561ApprovedInvestigationalWithdrawnValoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ).
Categories: Cellular and Gene TherapyDifenoxin
go.drugbank.com/drugs/DB01501ApprovedIllicitDifenoxin is a 4-phenylpiperidine which is closely related to the opioid analgesic meperidine. Difenoxin alone is a USA Schedule I controlled drug, as it may be habit forming. However, it is listed as a Schedule IV controlled drug if com...
Cobicistat
go.drugbank.com/drugs/DB09065ApprovedInvestigationalIncreasing systemic exposure of anti-retrovirals (ARVs) without increasing dosage allows for better treatment outcomes and a decreased side effect profile. … Although it does not have any anti-HIV activity, cobicistat acts as a pharmacokinetic enhancer by inhibiting cytochrome P450 3A isoforms (CYP3A) and therefore increases the systemic exposure of coadministered
Probenecid
go.drugbank.com/drugs/DB01032ApprovedInvestigationalProbenecid has also been used to treat patients with renal impairment, and, because it reduces the renal tubular excretion of other drugs, has been used as an adjunct to antibacterial therapy.