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AT2220
go.drugbank.com/drugs/DB05200InvestigationalAT2220 has been shown to increase GAA activity in cell lines derived from Pompe patients and in transfected cells expressing misfolded forms of GAA. … It is a small molecule designed to act as a pharmacological chaperone that specifically binds, stabilizes, and facilitates the proper folding and trafficking of α-glucosidase (GAA).
Prussian blue
go.drugbank.com/drugs/DB06783ApprovedIt contains ferric hexacyanoferrate(II) in a cubic lattice crystal structure. … It is insoluble in water but also tends to form a colloid thus can exist in either colloidal or water-soluble form, and an insoluble form.
Synonyms: Ferric hexacyanoferrate(II), Iron(III) hexacyanoferrate(II)Categories: Compounds used in a research, industrial, or household setting, Ion Exchange ActivityFitusiran
go.drugbank.com/drugs/DB15002ApprovedInvestigational[L52860] Fitusiran was first approved by the FDA on March 28, 2025, as routine prophylaxis therapy to prevent or reduce the frequency of bleeding episodes associated with hemophilia A or B. … [L52865] By causing the degradation of antithrombin mRNA and promoting thrombin generation, fitusiran works to restore hemostasis in patients with hemophilia.[A273770]
Synonyms: -D-GALACTOPYRANOSYL)OXY)PENTANAMIDO)PROPYL)AMINO)-3-OXOPR, SMALL INTERFERING RNA (SIRNA) INHIBITING ANTITHROMBIN LIVER PRODUCTION: DUPLEX OF ((2S,4R)-1-(1-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-16,16-BIS((3-((3-(5-((2-ACETAMIDO-2-DEOXY-.BETA.Categories: Antisense Elements (Genetics)Reslizumab
go.drugbank.com/drugs/DB06602ApprovedInvestigationalPatients often experience persistent or exacerbating symptoms overtime despite conventional first-line therapies available. … Approved by the FDA in March 2016, reslizumab is marketed under the brand name Cinqair for intravenous injection. It is injected once every four weeks via intravenous infusion.
Categories: Interleukin-5 AntagonistAlginic acid
go.drugbank.com/drugs/DB13518ApprovedInvestigationalWithdrawnAlginic acid is a linear polymer consisted of L-glucuronic acid and D-mannuronic acid residues connected via 1,4-glycosidic linkages [A32961]. … Available in different types of salt, alginic acid has been used in a variety of uses in food, cosmetics and pharmaceu-tical products for over 100 years [A32961].
Synonyms: (Alginate)n+1, (Alginate)nMixtures: GAVISCON® TABLETAS MASTICABLES SABOR A MENTA, MILPAX®NFGanaxolone
go.drugbank.com/drugs/DB05087ApprovedInvestigational[L41130] It was approved under the brand name ZTALMY by the US FDA in March 2022 for the treatment of seizures associated with CDKL5 deficiency disorder (CDD), becoming the first FDA-approved treatment … [L41135] In July 2023, ganaxolone was also approved under the same brand name and for the same indication by the EMA.[L47636]
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2B6 SubstratesThymol
go.drugbank.com/drugs/DB02513ApprovedInvestigationalA phenol obtained from thyme oil or other volatile oils. It is used as a stabilizer in pharmaceutic preparations. … It has been used for its antiseptic, antibacterial, and antifungal actions, and was formerly used as a vermifuge. (Dorland, 28th ed)
Synonyms: 5-Methyl-2-(1-Methylethyl)Phenol, 2-isopropyl-5-methylphenolMixtures: Marcs Original, Marcs Blue MintPonesimod
go.drugbank.com/drugs/DB12016ApprovedInvestigational[A232079] Ponesimod was granted FDA approval on 18 March 2021.[L32709] … Ponesimod is a selective sphingosine 1-phosphate receptor 1 modulator indicated in the treatment of relapsing forms of multiple sclerosis in adults.
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP3A SubstratesThyroid, porcine
go.drugbank.com/drugs/DB09100ApprovedInvestigational[A190831] Thyroid extract is no longer considered a first line therapy as it delivers a dose that is inconsistent with the stated strength of the tablet. … [A190831,L11755] Thyroid extract has been described in literature to treat hypothyroidism since 1891[A190807] but its use dates back as far as the 6th century.
Categories: Agents used to treat hypothyroidismFenretinide
go.drugbank.com/drugs/DB05076InvestigationalA synthetic retinoid that is used orally as a chemopreventive against prostate cancer and in women at risk of developing contralateral breast cancer. It is also effective as an antineoplastic agent.
Synonyms: N-(4-hydroxyphenyl)all-trans retinamide, 4-HPRCategories: Compounds used in a research, industrial, or household setting