dmd — TargetMol Chemicals

Cat No.	Product Name	Synonyms	Targets
T39516	Bocidelpar		PPAR
	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.		PPAR
T11062	dmDNA31		Others
	dmDNA31 is a rifamycin antibiotic that can inhibit the DNA-dependent RNA polymerase of bacteria and has strong bactericidal activity against Staphylococcus aureus.		Others
T15945	MA-0204		PPAR
	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...		PPAR
T3424	Ezutromid	BMN 195,VOX-C1100,SMT C1100	Others
	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 ...		Others
T38860	Golodirsen	Golodirsen,SRP-4053
	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).
T39517	Suvodirsen	WVE-210201
	Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).
T61645	Utrophin modulator 1
	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...
T66481	TG-693
	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...
T39455	Viltolarsen	NS-065,NCNP-01
	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 ...
T60594	OX01914
	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].
T14129	Adenylosuccinic acid	Aspartyl adenylate,Adenylosuccinate	Others
	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...		Others
T38859	Casimersen	SRP-4045
	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...
T73919	Adenylosuccinic acid tetraammonium
	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...
T41267	PROTAC(H-PGDS)-7
	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)-...
T75137	BAY-747
	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...
T37880	OPC-167832
	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...