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Cat. No.	Product Name	Target	Signaling Pathways
T39516	Bocidelpar	PPAR	DNA Damage/DNA Repair; Metabolism
	Bocidelpar 是一种有效的过氧化物酶体增殖物激活受体 delta 调节剂，可改善杜兴肌营养不良肌肉细胞中的线粒体生物合成和功能。
T7837	SR8278	Others	Others
	SR8278 是竞争性核血红素受体REV-ERB 合成拮抗剂，能够抑制 REV-ERBα 转录活性(EC50:0.47 μM)。它可用于调节生物体内的代谢和研究生物节律。
T17217	Vamorolone 地塞米松EP杂质E,VBP15	Glucocorticoid Receptor; NF-κB	Endocrinology/Hormones; NF-κB
	Vamorolone (VBP15) 是一种首创的，具有口服活性的解离性类固醇 (dissociative steroidal) 抗炎药和膜稳定剂。它抑制 (NF-κB) 抑制作用，并降低了激素的影响。它改善肌营养不良，无副作用。
T3424	Ezutromid BMN 195,VOX-C1100,依珠曲米,SMT C1100	Others	Others
	Ezutromid (BMN 195) 是一种具有口服活性的、首创的苯并恶唑 utrophin 调节剂，EC50=0.91 μM。它抑制人肝微粒体 CYP1A2 酶活性，IC50=5.4 μM。它可用于 Duchenne 型肌营养不良症 (DMD) 的研究。
T83633	1-N-Methyl-4-mercaptohistidine disulfide	Others	Others
	1-N-Methyl-4-mercaptohistidine disulfide 是一种潜在的杜氏菌叶绿体偶联因子1氧化还原调节剂，是氧化形式的卵硫醇A，抑制光触发的CF1 ATP酶活性。
T9821	ART-CHEM-BB B025267 2-(4-二乙基氨基苯基)-6-甲基-5-苯并三唑胺	Others	Others
	ART-CHEM-BB B025267 是 utrophin 生成的上调剂，EC50 为 1.8 μM，可用于治疗 Duchenne 肌营养不良症的研究。
T1805	Ataluren PTC124	CFTR; Autophagy	Autophagy; Membrane transporter/Ion channel
	Ataluren (PTC124) 是一种可口服的 CFTR-G542X 无义等位基因抑制剂。
T38554	Eteplirsen 依特立生；依特普森,AVI 4658
	Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research 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-skipped dystrophin protein.
T39517	Suvodirsen WVE-210201
	Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).
T14129	Adenylosuccinic acid Aspartyl adenylate,Adenylosuccinate	Others	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 muscular dystrophy(DMD)[1] and can be co
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.
T60594	OX01914
	OX01914 是一种水溶性和细胞膜可渗透的 utrophin 调节剂，能够上调 utrophin 蛋白水平，EC50值为 20.5 μM。 OX01914 可用于研究杜氏肌肉营养不良症(DMD)。
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).
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 dystrophy (DMD).
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 the myocardium and diaphragm. In a
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-established [1].
T37449	SR 12460
	SR 12460 is a mimetic of the IκB kinase β (IKKβ) NEMO/IKKγ-binding domain (NBD) that inhibits the protein-protein interaction between the IKK complex subunits NF-κB essential modulator (NEMO/IKKγ) and IKKβ.1It inhibits TNF-α-induced NF-κB activation in a reporter assay when used at concentrations ranging from 25 to 150 μM. It reduces LPS-induced production of IL-6 in RAW 264.7 cells. SR 12460 (30 mg/kg) improves grip strength in themdxmouse model of Duchenne muscular dystrophy. 1.Zhao, J., Zhang...
T75137	BAY-747
	BAY-747 (BAY 1165747) 为一种通过口服生效且具有脑渗透性的sGC刺激剂。它能可逆地逆转L-NAME所诱导的记忆障碍并在大鼠中增强认知功能。BAY-747还可在有意识状态下降低正常血压及自发性高血压大鼠(SHR)的血压。此外，BAY-747在mdx/mTRG2小鼠模型中改善了与Duchenne肌营养不良症(DMD)相关的骨骼肌功能。
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 frame by skipping exon 45. This action ultimately leads to the production of a functional dystrophin protein, albeit internally truncated. Casimersen finds utility in Duchenne muscular dystrophy (DMD) research.
T73919	Adenylosuccinic acid tetraammonium
	Adenylosuccinic acid tetraammonium（Adenylosuccinate; Aspartyl adenylate）是一种在核苷酸循环代谢中起作用的口服活性嘌呤核糖核苷单磷酸。该化合物可通过腺苷琥珀酸裂解酶转化为富马酸，展现出在杜氏肌营养不良症（DMD）研究中的应用潜力。

化合物

Cat.No: T39516

Target: PPAR

Cat.No: T7837

Target: Others

Cat.No: T17217

Synonym: 地塞米松EP杂质E,VBP15

Target: Glucocorticoid Receptor, NF-κB

Cat.No: T3424

Synonym: BMN 195,VOX-C1100,依珠曲米,SMT C1100

Target: Others

1-N-Methyl-4-mercaptohistidine disulfide

Cat.No: T83633

Target: Others

ART-CHEM-BB B025267

Cat.No: T9821

Synonym: 2-(4-二乙基氨基苯基)-6-甲基-5-苯并三唑胺

Target: Others

Cat.No: T1805

Synonym: PTC124

Target: CFTR, Autophagy

Cat.No: T38554

Synonym: 依特立生；依特普森,AVI 4658

Cat.No: T34425

Synonym: Read-through compound 13,RTC 13,RTC-13

Cat.No: T66481

Cat.No: T39517

Synonym: WVE-210201

Adenylosuccinic acid

Cat.No: T14129

Synonym: Aspartyl adenylate,Adenylosuccinate

Target: Others

Cat.No: T34426

Synonym: Read-through compound-14,RTC 14,RTC-14,Read-through compound 14

Cat.No: T60594

Cat.No: T38860

Synonym: Golodirsen,SRP-4053

Cat.No: T39455

Synonym: NS-065,NCNP-01

Cat.No: T33595

Synonym: AZD3582,Nitronaproxen,AZD-3582,Naproxen-N-butyl nitrate,HCT3012,AZD 3582

Utrophin modulator 1

Cat.No: T61645

Cat.No: T37449

Cat.No: T75137

Cat.No: T38859

Synonym: SRP-4045

Adenylosuccinic acid tetraammonium

Cat.No: T73919

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