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Displaying results 5661 - 5670 of 9887 in total
  1. Drug

    Nedosiran

    go.drugbank.com/drugs/DB17635
    ApprovedInvestigational

    Nedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate. Oxalate, particularly calcium oxalate, precipitation is the main cause of kidney stones formati...

  2. Drug

    Ammonium tetrathiomolybdate

    go.drugbank.com/drugs/DB17641
    Investigational

    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

    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]

  7. Drug

    ATX-F8-117

    go.drugbank.com/drugs/DB17712
    Experimental

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

  8. 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.

  9. 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]

  10. Drug

    mRNA-3351

    go.drugbank.com/drugs/DB17718
    Experimental

    mRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome