duchenne muscular dystrophy — 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
T15198	Edasalonexent	CAT-1004	NF-κB
	Edasalonexent (CAT-1004) is an orally available NF-κB inhibitor for the improvement of Duchenne muscular dystrophy.		NF-κB
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
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
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).
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...
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.
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
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...
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...
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].
T82534	Domagrozumab	PF-06252616
	Domagrozumab, an anti-myostatin humanized monoclonal antibody, exhibits a high affinity for human myostatin with a dissociation constant (K D) of 2.6 pM, promoting muscle anabolic activity. It is currently under investig...