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T39516 |
Bocidelpar
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PPAR
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Bocidelpar is a modulator of PPARδ with an EC50 of 7.80 nM and improves mitochondrial biogenesis and function in Duchenne Muscular Dystrophy muscle cells. |
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T11062 |
dmDNA31
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Others
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dmDNA31 is a rifamycin antibiotic that can inhibit the DNA-dependent RNA polymerase of bacteria and has strong bactericidal activity against Staphylococcus aureus. |
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T15945 |
MA-0204
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PPAR
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MA-0204 is a highly selective and orally available peroxisome proliferator-activated receptor δ (PPARδ) modulator (EC50s: 0.4 nM, 7.9 nM and 10 nM for human, mouse and rat PPARδ, respectively). It is a potential treatmen... |
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T3424 |
Ezutromid
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BMN 195,VOX-C1100,SMT C1100 |
Others
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Ezutromid (BMN 195) is a translation modulator of Small utrophin (EC50: 0.4 uM). Ezutromid is an orally bioavailable small molecule that transcriptionally upregulates the utrophin gene, increasing both utrophin mRNA and ... |
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T38860 |
Golodirsen
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Golodirsen,SRP-4053 |
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Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD). |
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T39517 |
Suvodirsen
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WVE-210201 |
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Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD). |
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T66481 |
TG-693
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TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skip... |
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T61645 |
Utrophin modulator 1
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Utrophin Modulator 1 (UM1) is a highly effective agent that upregulates utrophin protein levels, displaying an EC 50 of 0.11 μM. Its application in the investigation of Duchenne Muscular Dystrophy (DMD) has been well-est... |
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T39455 |
Viltolarsen
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NS-065,NCNP-01 |
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Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically targets the splicing of exon 53 in the dystrophin gene. It is utilized in research related to Duchenne muscular ... |
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T60594 |
OX01914
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OX01914 is a water-solube and permeable modulator of utrophin that can upregulate utrophin protein levels with an EC 50 of 20.5 μM. OX01914 can be used in Duchenne muscular dystrophy (DMD) study [1]. |
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T14129 |
Adenylosuccinic acid
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Aspartyl adenylate,Adenylosuccinate |
Others
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Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid has the potential for the study of duchenne muscu... |
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T38859 |
Casimersen
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SRP-4045 |
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Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading fra... |
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T73919 |
Adenylosuccinic acid tetraammonium
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Adenylosuccinic Acid Tetraammonium (also known as Adenylosuccinate or Aspartyl Adenylate) is an orally active compound that functions as a purine ribonucleoside monophosphate and is involved in nucleotide cycle metabolis... |
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T41267 |
PROTAC(H-PGDS)-7
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PROTAC(H-PGDS)-7 is a selective and potent small molecule hematopoietic prostaglandin D synthase (H-PGDS) PROTAC degradation agent that exhibits degradation activity in KU812 cells with a DC50 of 17.3 pM. PROTAC(H-PGDS)-... |
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T75137 |
BAY-747
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BAY-747 (BAY 1165747) is an orally administered, brain-penetrant compound that stimulates soluble guanylate cyclase (sGC). It effectively counteracts memory impairments induced by L-NAME and improves cognitive performanc... |
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T37880 |
OPC-167832
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OPC-167832 is a potent and orally active dprE1 Inhibitor with an IC50 of 0.258 μM. OPC-167832 has antituberculosis activity and can be used for the research of tuberculosis caused by Mycobacterium tuberculosis[1].
OPC-16... |
