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Results for "

muscular dystrophy

" in MCE Product Catalog:

17

Inhibitors & Agonists

1

Natural
Products

Cat. No. Product Name Target Research Areas
  • HY-132592
    Suvodirsen

    WVE-210201

    Others Metabolic Disease
    Suvodirsen (WVE-210201) is a oligonucleotide. Suvodirsen has the potential for study Duchenne muscular dystrophy (DMD).
  • HY-108753
    Eteplirsen

    AVI 4658

    Others Metabolic Disease
    Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research.
  • HY-132585
    SRP-5051

    Vesleteplirsen

    Others Others
    SRP-5051 is a next-generation antisense oligonucleotide of peptide phosphorodiamidate morpholino oligomer (PPMO). SRP-5051 targeting exon 51 skipping in Duchenne muscular dystrophy (DMD).
  • HY-132611
    Golodirsen

    SRP-4053

    Others Others
    Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD).
  • HY-127137A
    Adenylosuccinic acid tetraammonium

    Adenylosuccinate tetraammonium; Aspartyl adenylate tetraammonium

    Endogenous Metabolite Metabolic Disease
    Adenylosuccinic acid tetraammonium (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid tetraammonium can be converted into fumaric acid through adenylosuccinate lyase. Adenylosuccinic acid tetraammonium has the potential for the study of duchenne muscular dystrophy(DMD).
  • HY-109017
    Vamorolone

    VBP15

    Glucocorticoid Receptor Mineralocorticoid Receptor NF-κB Inflammation/Immunology
    Vamorolone (VBP15) is a first-in-class, orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone shows potent NF-κB inhibition and substantially reduces hormonal effects.
  • HY-132586
    Viltolarsen

    NS-065/NCNP-01

    Others Others
    Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD).
  • HY-17614
    Ezutromid

    SMT C1100; BMN 195; VOX-C1100

    Others Others
    Ezutromid (SMT C1100) is a first-in-class, orally active benzoxazole utrophin modulator with an EC50 of 0.91 μM. Ezutromid can be used for the research Duchenne muscular dystrophy (DMD). Ezutromid inhibits CYP1A2 enzymic activity in human liver microsomes (HLM) with an IC50 of 5.4 μM.
  • HY-134377
    Bocidelpar

    PPAR Metabolic Disease
    Bocidelpar is a modulator of peroxisome proliferator-activated receptor delta (PPAR-δ). Bocidelpar improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy (DMD) muscle cells (extracted from patent WO2017062468A1, compound 2b).
  • HY-16361A
    Omigapil maleate

    CGP3466B maleate

    Apoptosis Metabolic Disease Neurological Disease
    Omigapil maleate, an orally bioavailable GAPDH nitrosylation inhibitor, abrogates Aβ1-42-induced tau acetylation, memory impairment, and locomotor dysfunction in mice. Omigapil maleate has the potential for the research of Alzheimer's disease. Omigapil maleate (CGP3446B maleate) is a apoptosis inhibitor. Omigapil maleate can be used for the research of congenital muscular dystrophy (CMD).
  • HY-109134
    Pizuglanstat

    Prostaglandin Receptor Metabolic Disease
    Pizuglanstat (compound 3) is a prostaglandin D synthase inhibitor with an IC50 of 76 nM for human hematopoietic prostaglandin D synthases (H-PGDS). Pizuglanstat can be used for myodegenerative disease research, such as muscular dystrophy.
  • HY-145724
    Drisapersen

    Kyndrisa; GSK2402968A; PRO051

    Others Others
    Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
  • HY-18102
    GLPG0492

    Androgen Receptor Others
    GLPG0492 is a non-steroidal selective androgen receptor modulator (potency 12 nM). GLPG0492 has the potential for the research of musculo-skeletal diseases such as sarcopenia and cachexia.
  • HY-114739
    MA-0204

    PPAR Metabolic Disease
    MA-0204 is a potent, highly selective and orally available peroxisome proliferator activated receptor δ (PPARδ) modulator with EC50s of 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively. Potential treatment for Duchene Muscular Dystrophy (DMD).
  • HY-132584
    Casimersen

    SRP-4045

    Others Others
    Casimersen (SRP-4045) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer subclass. Casimersen binds to exon 45 of dystrophin pre-mRNA, restores the open-reading frame (by skipping exon 45) resulting in the production of an internally truncated but functional dystrophin protein. Casimersen can be used for the research of Duchenne muscular dystrophy (DMD).
  • HY-136340
    21-Acetoxypregna-1,4,9(11),16-tetraene-3,20-dione

    Glucocorticoid Receptor NF-κB Metabolic Disease Inflammation/Immunology
    21-Acetoxypregna-1,4,9(11),16-tetraene-3,20-dione is an intermediate of delta 9,11 steroids synthesis, for example, Vamorolone (HY-109017). The delta 9,11 steroids are modifications of glucocorticoids and has anti-inflammatory properties. The delta 9,11 steroids are agents for protection against cell damage (lipid peroxidation) and inhibition of neovascularization.
  • HY-147332
    TCL053

    Others Neurological Disease
    TCL053 is an ionizable lipid carrier and used to introduce active components, in particular nucleic acids, into cells with excellent efriciency. TCL053, together with DPPC (Dipalmitoylphosphatidylcholine), PEG-DMG (Polyethylene glycoldimyristoyl glycerol), and cholesterol, forms lipid nanoparticle (LNP) which is able to deliver Cas9 mRNA and sgRNA into skeletal muscle.