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Displaying results 5441 - 5450 of 9118 in total
  1. Drug

    BT200

    go.drugbank.com/drugs/DB18333
    Investigational

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

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

    AVR-RD-04

    go.drugbank.com/drugs/DB18644
    Experimental

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

    Synonyms: autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin
  3. 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).

  4. Drug

    Binetrakin

    go.drugbank.com/drugs/DB12182
    Investigational

    Binetrakin has been used in trials studying the treatment of HIV Infections, Sarcoma, Kaposi, Non-Hodgkin's Lymphoma (NHL), Myelodysplastic Syndrome (MDS), and Leukemia, Acute Myelogenous (AML), among

  5. Drug

    Mavoglurant

    go.drugbank.com/drugs/DB13004
    Investigational

    Mavoglurant has been used in trials studying the treatment of Patient Diagnosed With OCD and and Resistant to SSRI Treatment (Failed SSRI Over 12 Weeks at Appropriate Doses).

  6. Drug

    Propanidid

    go.drugbank.com/drugs/DB13234
    Experimental

    An intravenous anesthetic that has been used for rapid induction of anesthesia and for maintenance of anesthesia of short duration. (From Martindale, The Extra Pharmacopoeia, 30th ed, p918)

  7. Drug

    Utidelone

    go.drugbank.com/drugs/DB17575
    Investigational

    Utidelone (UTD1), a novel microtubule stabilizing agent, is an epothilone B analogue produced by genetic engineering.[A257544,A257549] It is under investigation in clinical trials for various cancers.

  8. Drug

    INZ-701

    go.drugbank.com/drugs/DB17629
    Investigational

    INZ-701 is an enzyme replacement therapy under development with the intention to be used for the treatment of calcification disorders of the circulatory system, bones, and kidneys.

  9. Drug

    mRNA-3705

    go.drugbank.com/drugs/DB17668
    Investigational

    mRNA-3705 is an investigational mRNA encoding methylmalonic CoA mutase (MUT) enzyme. Developed by Moderna TX Inc., it is being investigated for the treatment of methylmalonic academia.[A258843]

  10. Drug

    Efdoralprin alfa

    go.drugbank.com/drugs/DB18384
    Investigational

    Efdoralprin alfa is a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein.[L54928] It is being investigated as a restorative therapy in adults with AATD emphysema.[L54928]