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Displaying results 8531 - 8540 of 10000 in total
  1. Drug

    Zodasiran

    go.drugbank.com/drugs/DB17626
    Investigational

    It was granted an orphan drug designation for the treatment of homozygous familial hypercholesterolemia (HoFH).[L45869] … ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin-like protein 3 (ANGPTL3).

  2. Drug

    BT200

    go.drugbank.com/drugs/DB18333
    Investigational

    [A261491] It is under investigation for the treatment of von Willebrand disease and hemophilia A.[L48156] … BT200 (Rondoraptivon pegol) is a PEGylated anti-von Willebrand factor (VWF) aptamer targeted against the VWF A1 domain.

    Synonyms: PEGylated aptamer targeting Von Willebrand Factor A1 domain
  3. Drug

    AVR-RD-04

    go.drugbank.com/drugs/DB18644
    Experimental

    It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin. … AVR-RD-04 is an investigational gene therapy for cystinosis.

    Synonyms: autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin
  4. Drug

    EGT-201

    go.drugbank.com/drugs/DB17648
    Experimental

    EGT-201 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the alpha-N-acetyglucosaminidase (NAGLU) transgene.

  5. Drug

    MD05

    go.drugbank.com/drugs/DB06375
    Investigational

    MD05 is a beta-tricalcium phosphate (beta-TCP) coated with recombinant human growth/differentiation factor-5 (rhGDF-5).

  6. Drug

    SYNB1353

    go.drugbank.com/drugs/DB17666
    Investigational

    SYNB1353 is a strain of the probiotic bacteria _Escherichia coli_ (_E. coli_) type Nissle and an engineered probiotic drug. … [A258833] Developed by Synlogic Operating Company, Inc., SYNB1353 is being investigated for the treatment of homocystinuria.

  7. Drug

    Indenolol

    go.drugbank.com/drugs/DB08952
    ApprovedWithdrawn

    Indenolol is a drug of the beta-adrenergic blocker class.

  8. Drug

    Bos taurus skin

    go.drugbank.com/drugs/DB10406
    Approved

    Bos taurus skin or pelt is used in allergenic testing.

  9. Drug

    Mesocricetus auratus skin

    go.drugbank.com/drugs/DB10605
    Approved

    Mesocricetus auratus skin or pelt is used in allergenic testing.

  10. Drug

    Iodide

    go.drugbank.com/drugs/DB12754
    ApprovedInvestigationalWithdrawn

    Iodide has been investigated for the treatment of Goiter, Nodular.