duchenne — 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
T7837	SR8278		Others
	SR8278 is an REV-ERBα antagonist(EC50 = 0.47 µM), blocking activation of the receptor by the synthetic agonist GSK 4112		Others
T17217	Vamorolone	VBP15	Glucocorticoid Receptor , NF-κB
	Vamorolone (VBP15) is an orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer. Vamorolone improves muscular dystrophy without side effects. Vamorolone displays effective NF-κB inhibition an...		Glucocorticoid Receptor , NF-κB
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
T9821	ART-CHEM-BB B025267		Others
	ART-CHEM-BB B025267 is the upregulator of utrophin production with EC50 of 1.8 μM and can be used in research on the treatment of Duchenne muscular dystrophy.		Others
T1805	Ataluren	PTC124	CFTR , Autophagy
	Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and olde...		CFTR , Autophagy
T83633	1-N-Methyl-4-mercaptohistidine disulfide		Others
	1-N-Methyl-4-mercaptohistidine disulfide is a potential Duchenne chloroplast coupling factor 1 redox regulator, an oxidized form of ochratoxin A, that inhibits light-triggered CF1 ATPase activity.		Others
T38554	Eteplirsen	AVI 4658
	Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy.
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).
T34426	RTC14	Read-through compound-14,RTC 14,RTC-14,Read-through compound 14
	RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
T34425	RTC13	Read-through compound 13,RTC 13,RTC-13
	RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
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 ...
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...
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...
T33595	Naproxcinod	AZD3582,Nitronaproxen,AZD-3582,Naproxen-N-butyl nitrate,HCT3012,AZD 3582
	Naproxcinod, a nitric oxide-releasing NSAID derivative, significantly improved skeletal muscle strength and fatigue resistance in sedentary and exercise mice, reducing inflammatory infiltration and fibrotic deposition in...
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].
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...