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Displaying results 2891 - 2900 of 10000 in total
  1. Drug

    ENTR-601-44

    go.drugbank.com/drugs/DB17836
    Investigational

    ENTR-601-44 is an exon 44 skipping phosphorodiamidate morpholino oligonucleotide (PMO). Developed by Entrada Therapeutics, it was investigated for the treatment of Duchenne muscular dystrophy.[L46641]

  2. Drug

    DS-1211

    go.drugbank.com/drugs/DB17837
    Investigational

    DS-1211 is a potent and selective novel small molecule tissue-nonspecific alkaline phosphatase (TNAP) inhibitor.[A259786]

    Synonyms: N-Acetyl-D-leucine, D-leucine, N-acetyl-
  3. Drug

    OAV201

    go.drugbank.com/drugs/DB17842
    Experimental

    OAV201 is a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter

  4. Drug

    GS030-DP

    go.drugbank.com/drugs/DB17847
    Investigational

    It is a component of GS030, an investigational optogenetic treatment, in combination with a medical device GS030-MD.[A259816, L46701] … GS030-DP is an optogenetic gene therapy being investigated for the treatment of retinitis pigmentosa.

  5. Drug

    AAV1-FS344

    go.drugbank.com/drugs/DB17855
    Investigational

    AAV1-FS344 is an investigational gene therapy developed by Milo Biotechnology to deliver follistatin, a potent myostatin inhibitor. … It is being investigated for the treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and inclusion body myositis.[L46742, L46747, L46752]

  6. Drug

    Soraprazan

    go.drugbank.com/drugs/DB17856
    Experimental

    Synonyms: (7r,8r,9r)-7-(2-methoxyethoxy)-2,3-dimethyl-9-phenyl-7,8,9,10-tetrahydroimidazo(1,2-h)(1,7)naphtyridin-8-olCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-Ring
  7. Drug

    SAR-439459

    go.drugbank.com/drugs/DB17864
    Investigational

    SAR-439459 is a pan-transforming Growth Factor-beta (TGFβ) neutralizing antibody.[A259862] Developed by Sanofi, it is being investigated for the treatment of cancers and Osteogenesis Imperfecta.

  8. Drug

    QRX-411

    go.drugbank.com/drugs/DB17868
    Experimental

    It addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the _USH2A_ gene.[L46777] … QRX-411 is a first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of the functional USH2A protein.

  9. Drug

    MANF

    go.drugbank.com/drugs/DB17885
    Experimental

    MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.

  10. Drug

    GIVI-MPC

    go.drugbank.com/drugs/DB17905
    Experimental

    GIVI-MPC is a therapy that uses human-induced pluripotent stem cell-derived myogenic progenitor cells.