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Vestronidase alfa
go.drugbank.com/drugs/DB12366ApprovedInvestigationalIn clinical trials, vestronidase alfa treatment demonstrated improvement and stabilization in motor symptoms by increasing the patients' ability to walk longer distances in comparison to treatment with … Last two conditions are leading causes of fatalities in patients with MPS VII.
Sodium oxybate
go.drugbank.com/drugs/DB09072ApprovedInvestigationalin May 2023. … [L46302] In some countries, sodium oxybate has been investigated and used in alcohol withdrawal syndrome (AWS) to aid abstinence maintenance in alcohol use disorders.[A251440, A251445]
Penicillamine
go.drugbank.com/drugs/DB00859ApprovedInvestigationalIt is an α-amino acid metabolite of penicillin, although it has no antibiotic properties.
Categories: Compounds used in a research, industrial, or household settingPalovarotene
go.drugbank.com/drugs/DB05467ApprovedTreatment options for patients with FOP are extremely limited, although there has been substantial recent interest in novel treatments for this disease. … [A244920] Palovarotene was approved for use in Canada in January 2022 for the management of heterotopic ossification (HO) in patients with FOP, representing the first global approval for any FOP therapy
Vanzacaftor
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.
Endometrial Hyperplasia
go.drugbank.com/conditions/DBCOND0001317Synonyms: Complex endometrial hyperplasia (disorder)Tegafur
go.drugbank.com/drugs/DB09256ApprovedInvestigationalIt is a pyrimidine analogue used in combination therapies as an active chemotherapeutic agent in conjunction with [DB09257] and [DB03209], or along with [DB00544] as [DB09327]. … When converted and bioactivated to 5-FU, the drug mediates an anticancer activity by inhibiting thymidylate synthase (TS) during the pyrimidine pathway involved in DNA synthesis. 5-FU is listed on the
International brands: ESUEEW AN TEthoxzolamide
go.drugbank.com/drugs/DB00311ApprovedWithdrawnEthoxzolamide is a sulfonamide used as diuretic and in glaucoma. It inhibits carbonic anhydrase activity in proximal renal tubules to decrease reabsorption of water, sodium, potassium, bicarbonate.
Infliximab
go.drugbank.com/drugs/DB00065ApprovedInvestigationalIn clinical trials, multiple infusions of infliximab displayed in a reduction of signs and symptoms of inflammatory diseases and induction of remission in patients who have had an inadequate response to … Infliximab was first approved by the FDA in 1998 under the market name Remicade as an intravenous injection.
Categories: Complex MixturesProducts: Remsima 120mg solution for injection in pre-filled pen, REMSIMA SOLUTION FOR SUBCUTANEOUS INJECTION 120 MG/ML IN PRE-FILLED PENFerric maltol
go.drugbank.com/drugs/DB15598ApprovedInvestigationalFerric maltol is an iron(III) atom complexed with 3 maltol molecules to increase the bioavailability compared to iron(II), without depositing it in the duodenum as insoluble ferric hydroxide and phosphate … [A189291] Ferric maltol was initially granted FDA approval on July 25, 2019, for use in adults.