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Onasemnogene abeparvovec
go.drugbank.com/drugs/DB15528ApprovedInvestigational[Nusinersen] is another gene therapy that is currently approved by the FDA for the treatment of SMA in pediatric and adult patients. … [L9194] SMA is a rare genetic disease that affects the survival and function of motor neurons, leading to debilitating and often fatal muscle weakness.
Prademagene zamikeracel
go.drugbank.com/drugs/DB17895ApprovedInvestigational[L53008] Prademagene zamikeracel was first approved by the FDA on April 29, 2025, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a skin disorder associated with excessive blistering … [L53008] COL7A1 encodes type VII collagen (C7), which is a major component of anchoring fibrils (AF) that are critical for dermal-epidermal cohesion.
Damoctocog alfa pegol
go.drugbank.com/drugs/DB14700ApprovedInvestigationalIn order to extend half-lives, techniques such as fusion to protein conjugates (Fc part of IgG1 or albumin), chemical modification (PEGylation), and protein sequence modification have been utilized. … [A38907] Also known as, BAY94-9027, Damoctocog alfa pegol is a longer-acting Factor VIII therapy formulated with polyethylene glycol (PEG) to reduce the number of infusions necessary to prevent bleeds
Alatrofloxacin
go.drugbank.com/drugs/DB09335ApprovedWithdrawnAlatrofloxacin is a fluoroquinolone antibiotic developed by Pfizer, delivered as a mesylate salt. It was withdrawn from the U.S. market in 2001.
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.
Categories: Preparations With No Effect on Uric Acid Metabolism, Compounds used in a research, industrial, or household settingNortriptyline
go.drugbank.com/drugs/DB00540ApprovedInvestigational[L11878] It is used in the treatment of major depression and is also used off-label for chronic pain and other conditions.[L11881]
Categories: Antidepressive Agents Indicated for Depression, Serotonergic Drugs Shown to Increase Risk of Serotonin SyndromeRelugolix
go.drugbank.com/drugs/DB11853ApprovedInvestigational[L27996] In addition to its relative ease-of-use, relugolix was shown to be superior in the depression of testosterone levels when compared to [leuprolide], another androgen deprivation therapy used in … administration - and therefore provides a less burdensome therapeutic option for patients who might otherwise require clinic visits for administration by healthcare professionals.
Bexarotene
go.drugbank.com/drugs/DB00307ApprovedInvestigationalBexarotene (Targretin) is an antineoplastic agent indicated by the FDA for Cutaneous T cell lymphoma. It has been used off-label for lung cancer, breast cancer, and Kaposi's sarcoma.
Categories: Retinoids for cancer treatment, Compounds used in a research, industrial, or household settingCarglumic acid
go.drugbank.com/drugs/DB06775ApprovedInvestigationalCarglumic acid is a drug used for the treatment of hyperammonemia in patients with a deficiency in N-acetyl glutamate synthase. … Carglumic acid was approved by the U.S. Food and Drug Administration (FDA) on 18 March 2010.
Fluoride ion F-18
go.drugbank.com/drugs/DB09398ApprovedInvestigationalIts primary indication is for bone imaging. … It contains radioactive fluoride F 18 that is used for diagnostic purposes in conjunction with positron emission tomography (PET) imaging and is administered by intravenous injection.