2. Signaling Pathways
  3. Cytoskeleton
  4. Dystrophin
  5. Dystrophin Modulator

Dystrophin Modulator

Dystrophin Modulators (8):

Cat. No. Product Name Effect Purity
  • HY-132586A
    Viltolarsen sodium
    		Modulator
    Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research.
  • HY-132611
    Golodirsen
    		Modulator
    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-101459
    RTC13
    		Modulator 99.31%
    RTC13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy (DMD).
  • HY-122631
    TG693
    		Modulator 99.95%
    TG693 is an orally active inhibitor of CLK1. TG693 regulates the mutated exon 31 of the dystrophin gene in vivo. TG693 is used in Duchenne muscular dystrophy (DMD) research.
  • HY-132584
    Casimersen
    		Modulator
    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-132586
    Viltolarsen
    		Modulator
    Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research.
  • HY-163665
    AHR antagonist 8
    		Modulator
    AHR antagonist 8 (compound SG-02) is a regulator of utrophin, a homolog of dystrophin, and an AhR antagonist (Kd: 41.68 nM). Studies have shown that 800 nM of AHR antagonist 8 can upregulate utrophin by 2.7 times. AHR antagonist 8 also stimulates increased MyHC expression, suggesting that it has the potential to enhance myogenesis. After ADME evaluation, AHR antagonist 8 also has a certain oral bioavailability.
  • HY-123359
    RTC14
    		Modulator
    RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene.