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Cysteamine
go.drugbank.com/drugs/DB00847ApprovedInvestigationalCystinosis is a rare disease caused by mutations in the CTNS gene that encodes for cystinosin, a protein responsible for transporting cystine out of the cell lysosome. … [L15606] Cysteamine eye drops are a practical and effective option for those affected by ocular cystinosis.
Belatacept
go.drugbank.com/drugs/DB06681ApprovedInvestigationalThe drug has activity as a selective co-stimulation modulator with inhibitory activity on T lymphocytes. It is approved for the treatment of rheumatoid arthritis. … Structurally, abatacept is a glycosylated fusion protein with a MALDI-MS molecular weight of 92,300 Da and it is a homodimer of two homologous polypeptide chains of 357 amino acids each.
Triheptanoin
go.drugbank.com/drugs/DB11677ApprovedInvestigational[A214812,A214817] Complications in lc-FAOD patients are reduced from approximately 60% to approximately 10% with the addition of triheptanoin. … Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation.
Hydroxyamphetamine
go.drugbank.com/drugs/DB09352ApprovedHydroxyamphetamine is a derivative of amphetamines. Hydroxyamphetamine is intended mainly as local eye drops for diagnostic purposes. … Among the minor side effects from its use are: change in color vision, difficulty seeing at night, dry mouth, headache, increased sensitivity of eyes to sunlight, muscle stiffness or tightness and temporary
Linerixibat
go.drugbank.com/drugs/DB11729ApprovedInvestigationalLinerixibat is a first-in-class and small-molecule inhibitor of the ileal bile acid transporter (IBAT). … [A275481, A275482] On March 19, 2026, linerixibat was approved by the FDA for the treatment of cholestatic pruritus in patients with primary biliary cholangitis (PBC).
Diethylcarbamazine
go.drugbank.com/drugs/DB00711ApprovedInvestigationalVet approvedWithdrawnAn anthelmintic used primarily as the citrate in the treatment of filariasis, particularly infestations with Wucheria bancrofti or Loa loa.
Thalidomide
go.drugbank.com/drugs/DB01041ApprovedInvestigationalWithdrawnIt has been reintroduced and used for a number of inflammatory disorders and cancers. … A piperidinyl isoindole originally introduced as a non-barbiturate hypnotic, thalidomide was withdrawn from the market due to teratogenic effects.
International brands: Pro-ban MSulfur hexafluoride
go.drugbank.com/drugs/DB11104ApprovedInvestigationalin adult patients with suboptimal echocardiograms •in ultrasonography of the liver for characterization of focal liver lesions in adult and pediatric patients … Sulfur hexafluoride is an ultrasound contrast agent indicated for use •in echocardiography to opacify the left ventricular chamber and to improve the delineation of the left ventricular endocardial border
Products: SONOVUE POWDER FOR DISPERSION FOR INJECTION 8 microlitres/mlIodide
go.drugbank.com/drugs/DB12754ApprovedInvestigationalWithdrawnIodide has been investigated for the treatment of Goiter, Nodular.
ATG-Fresenius S
go.drugbank.com/drugs/DB05320InvestigationalATG-Fresenius S is a concentrated anti-human T-lymphocyte immunoglobulin preparation derived from rabbits after immunization with a T-lympoblast cell line. … ATG-Fresenius S is an immunosuppressive product for the prevention and treatment of acute rejection following organ transplantation.