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  1. Drug

    Tofersen

    go.drugbank.com/drugs/DB14782
    ApprovedInvestigational

    [L46108] Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented. … [A259028,A259033] Tofersen was granted accelerated approval from the FDA on April 25, 2023, as the first treatment for adults with ALS caused by SOD1 mutation.

    Synonyms: DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP, -(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R
  2. Drug

    Tipapkinogene sovacivec

    go.drugbank.com/drugs/DB06239
    Investigational

  3. Drug

    Daprodustat

    go.drugbank.com/drugs/DB11682
    ApprovedInvestigational

    Daprodustat is a small-molecule hypoxia-inducible factor (HIF) prolyl hydroxylase (PHD) inhibitor that was developed by GSK. … [L43857] On February 1, 2023, daprodustat was fully approved by the FDA as the first oral treatment for anemia caused by chronic kidney disease in patients on dialysis.

    Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2C8 InhibitorsSynonyms: Glycine, N-((1,3-dicyclohexylhexahydro-2,4,6-trioxo-5-pyrimidinyl)carbonyl)-
  4. Drug

    Tipiracil

    go.drugbank.com/drugs/DB09343
    ApprovedInvestigational

    Tipiracil is a thymidine phosphorylase inhibitor. It is used in combination with trifluridine, in a ratio of 1:0.5, to form TAS-102. … The main function of Tipiracil in TAS-102 is to increase trifluridine bioavailability by inhibiting its catabolism.

    Mixtures: LONSURF FILM-COATED TABLET 20 MG/8.19 MG, LONSURF FILM-COATED TABLET 20 MG/8.19 MGCategories: MATE 1 Substrates with a Narrow Therapeutic Index, MATE 1 Substrates
  5. Drug

    Piperonyl butoxide

    go.drugbank.com/drugs/DB09350
    ApprovedVet approved

    Piperonyl butoxide is a semisynthetic derivative of safrole. … Piperonyl butoxide (PBO) is an organic compound used as a component of pesticide formulations. It is used for the treatment of head, pubic (crab), and body lice. Piperonyl butoxide is a synergist.

    Mixtures: KWELL-P %0,3 + %3 MEDİKAL ŞAMPUAN, 60 ML, KWELL-P %0,3 + %3 MEDİKAL ŞAMPUAN, 120 MLCategories: Cytochrome P-450 CYP1A2 Inhibitors, Cytochrome P-450 CYP3A Inhibitors
  6. Drug

    Berdazimer

    go.drugbank.com/drugs/DB18712
    Approved

    This decision is based on positive results demonstrated in 2 Phase 3 trials, B-SIMPLE 4 and B-SIMPLE 2, where reduced lesion counts were observed with once-a-day uses of berdazimer.[L49520] … Berdazimer is a polymeric substance consisting of a polysiloxane backbone (Si-O-Si bonds) with covalently bound N-diazeniumdiolate nitric oxide (NO) donors.

    Synonyms: Silsesquioxanes, 3-(2-hydroxy-1-methyl-2-nitrosohydrazinyl)propyl 3-(methylamino)propyl, polymers with silicic acid (h4sio4) tetra-et ester, hydroxy-terminated
  7. Pathway

    Hyperphenylalaninemia Due to Guanosine Triphosphate Cyclohydrolase Deficiency

    smpdb.ca/view/SMP0125583
    Disease

    Hyperphenylalaninemia is the high presence of phenylalanine in the system/blood caused by a genetic mutation. In this case a missense error in the gene which encodes GTP cyclohydrolase. Consequently, this form of hyperphenylalaninemia is...

    Drugs: Phenylalanine · Tyrosine · NADH · Sapropterin · Iron · Zinc +3 more
    Enzymes: Y+L amino acid transporter 1
  8. Drug

    Kappadione

    go.drugbank.com/drugs/DB09332
    Approved

    Kappadione is a Vitamin K derivative (chemically, it is menadiol sodium diphosphate), previously approved by FDA prior to 1982 and marketed by Lilly Marketing for this drug has been discontinued and is … Studies involving the active metabolite of this formulation, menadione, showed oocyte toxicity in a study of mice [L1544].

  9. Drug

    Methylcellulose

    go.drugbank.com/drugs/DB11228
    ApprovedInvestigational

    The Degree of Substitution (DS) of a given form of methyl cellulose is defined as the average number of substituted hydroxyl groups per glucose with a theoretical maximum of 3, however more typical values … Methyl cellulose is a hydrophilic white powder in pure form and dissolves in cold (but not in hot) water, forming a clear viscous solution or gel.

    Salts: Methylcellulose (25 MPa.s), Methylcellulose (100 MPa.s)Categories: Compounds used in a research, industrial, or household setting
  10. Drug

    Amlexanox

    go.drugbank.com/drugs/DB01025
    ApprovedWithdrawn

    Recurrent aphthous ulcer (RAU) is the most prevalent oral mucosal disease in humans, estimated to affect between 5% and 50% of the general population. … Amlexanox as a topical paste is a well tolerated treatment of recurrent aphthous ulcers.

    Categories: Heterocyclic Compounds, 1-RingSynonyms: 2-Amino-7-isopropyl-5-oxo-5H-(1)benzopyrano(2,3-b)pyridine-3-carboxylic acid