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Displaying results 1261 - 1270 of 10000 in total
  1. Drug

    Nedosiran

    go.drugbank.com/drugs/DB17635
    ApprovedInvestigational

    This approval is based on the favorable results from the pivotal phase 2 PHYOX<sup>TM</sup>2 and interim data from the ongoing phase 3 PHYOX<sup>TM</sup>3 clinical trials.[L48325] … hyperoxaluria type 1 (PH1) and relatively preserved kidney function.

  2. Drug

    Ammonium tetrathiomolybdate

    go.drugbank.com/drugs/DB17641
    Investigational

    Synonyms: Molybdate(2-), tetrathioxo-, ammonium (1:2), (t-4)-, Molybdate(2-), tetrathioxo-, diammonium, (t-4)-Categories: Compounds used in a research, industrial, or household setting
  3. Drug

    TAK-611

    go.drugbank.com/drugs/DB17644
    Investigational

    TAK-611, formerly known as SHP611, is a recombinant human arylsulfatase A. First developed by Takeda, it is being investigated for the treatment of metachromatic leukodystrophy.

  4. Drug

    AVR-RD-01

    go.drugbank.com/drugs/DB17651
    Investigational

    It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence.

  5. Drug

    mRNA-3927

    go.drugbank.com/drugs/DB17669
    Investigational

    mRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC) encapsulated within a lipid

  6. Drug

    HMI-103

    go.drugbank.com/drugs/DB17684
    Investigational

    HMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.

  7. Drug

    SerpinPC

    go.drugbank.com/drugs/DB17698
    Investigational

    SerpinPC is a mutated alpha-1-antitrypsin (α1AT) and serine protease inhibitor being investigated for the treatment of hemophilia.[A258993]

  8. Drug

    ATX-F8-117

    go.drugbank.com/drugs/DB17712
    Experimental

    Developed by Apitope, it is being investigated for the treatment of hemophilia A.[L46163]

  9. Drug

    Thalagen

    go.drugbank.com/drugs/DB17714
    Experimental

    Thalagen is a branded product developed by San Rocco Therapeutics. … It consists of autologous CD34-positive hematopoietic progenitor cells transduced with TNS9.3.55, a lentiviral vector encoding the normal human beta-globin gene.

  10. Drug

    MOD-5014

    go.drugbank.com/drugs/DB17715
    Investigational

    MOD-5014 is a long-acting carboxy-terminal peptide (CTP)-modified Factor IIa.[A259088] It is being investigated for the treatment of hemophilia.[L46173]