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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。
T11553	Heptamidine dimethanesulfonate SBi4211 dimethanesulfonate	Others	Others
	Heptamidine dimethanesulfonate (SBi4211 dimethanesulfonate) 是一种与喷他脒有关的、强效的钙结合蛋白 S100B 的抑制剂 (Kd=6.9 μM)，选择性地杀死表达 S100B 的黑素瘤细胞。它是有效的强直性肌营养不良 (DM) 研究工具。
T11861	LM11A-31 dihydrochloride	Others	Others
	LM11A-31 dihydrochloride 是一种具有高血脑屏障通透性的氨基酸衍生物，能阻断 p75 介导的细胞死亡。它是一种 p75NTR(神经营养蛋白受体 p75) 非肽调节剂，是口服有效的 proNGF 拮抗剂。它可以逆转中晚期的阿尔茨海默氏病小鼠模型中的胆碱能神经突营养不良。
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。
T13491	Neuromuscular-targeting compound 1	Others	Others
	Neuromuscular-targeting compound 1 可用于研究肌肉萎缩和神经肌肉相关疾病。
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。
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。
T72073	AKOS037652256
	AKOS037652256可作为 TRPML 调节剂，可用于治疗如溶酶体积存病、肌肉萎缩症、与年龄相关的常见神经退行性疾病、氧化应激或活性氧(ROS)相关疾病以及衰老等与 TRPML 活性相关的疾病。
T72072	2-benzylsulfanyl-6-methoxy-4-methylquinazoline	ROS	Immunology/Inflammation
	2-benzylsulfanyl-6-methoxy-4-methylquinazoline 可作为TRPML 调节剂，可用于治疗与TRPML 活性相关的疾病，如溶酶体储存病、肌肉萎缩症、氧化应激或活性氧(ROS)相关疾病和衰老。
T15945	MA-0204	PPAR	DNA Damage/DNA Repair; Metabolism
	MA-0204 是一种高选择性，可口服的过氧化物酶体增殖物激活受体 δ (PPARδ) 调节剂，对于人，小鼠和大鼠 PPARδ 的 EC50分别为 0.4、7.9 和 10 nM。它有用于 Duchene 肌营养不良症 (DMD) 的研究潜力。
T21792	CGP 3466B maleate 马来酸CGP3466B,Omigapil (Maleate),Omigapil maleate	Apoptosis	Apoptosis
	CGP 3466B maleate (Omigapil maleate) 是一种口服有效的 GAPDH 亚硝基化抑制剂，可消除 Aβ1-42 诱导的小鼠 tau 乙酰化，记忆障碍和运动功能障碍，Omigapil maleate 具有研究阿尔茨海默症的潜力。Omigapil maleate (CGP3446B maleate) 是一种有效的凋亡 (apoptosis) 抑制剂。Omigapil maleate 可用于研究先天性肌营养不良 (CMD)。
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.
T39517	Suvodirsen WVE-210201
	Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).
T79918	α7β1 integrin modulator-1	Integrin	Cytoskeletal Signaling
	α7β1integrinmodulator-1，作为一种高效的α7β1integrin调节剂，展现出在肌营养不良症研究中的应用潜力。
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.
T61182	(R)-BDP9066
	(R)-BDP9066 is a highly effective inhibitor of myotonic dystrophy kinase-related Cdc42-binding kinase (MRCK), effectively impeding cancer cell invasion, making it an invaluable resource for studying and researching proliferative diseases, particularly cancer.
T11553L	Heptamidine SBi4211	Others	Others
	Heptamidine is an effective Pentamidine-related inhibitor of the calcium-binding protein S100B (Kd: 6.9 μM). It selectively kills melanoma cells with S100B over those without S100B. It is a useful tool for the investigation of Myotonic dystrophy.
T21427	Mexiletine Mexitil,Mexiletene,KO-1173,KO1173,KO 1173
	Mexiletine is a non-selective voltage-gated sodium channel blocker. It is a medicine in the class of IB anti-arrhythmic. Mexiletine may also be used in patients experiencing refractory pain and is effective to treat muscle stiffness resulting from myotonic dystrophy or non-dystrophic myotonias.
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).
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.
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).
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].
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)。
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
T83175	Activated DPG Subunit
	Activated DPG Subunit 用于合成可诱导外显子 51 跳跃的外显子跳跃寡聚物偶联物。这些偶联物与人抗肌萎缩蛋白基因中选定的靶位点互补，有助于肌肉萎缩症的研究。
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
T82222	H-D-MeAla-EtVaI-VaI-MeLeu-AIa-D-AIa-MeLeu-MeLeu-MeVaI-MeBmt(OAc)-Abu-O-CH2-CH2-NHMe
	H-D-MeAla-EtVal-Val-MeLeu-AIa-D-AIa-MeLeu-MeLeu-MeVal-MeBmt(OAc)-Abu-O-CH2-CH2-NHMe 是一种非免疫抑制性的环孢菌素A衍生物，具备研究先天性肌营养不良症潜力[1]。
T83176	Activated C Subunit
	Activated C Subunit 用于合成可诱导外显子51跳跃的外显子跳跃寡聚物偶联物。所得寡聚物偶联物与人类抗肌萎缩蛋白基因特定靶位点互补，有助于研究肌肉萎缩症。
T73321	CLK1-IN-2
	CLK1-IN-2 是代谢稳定的Clk1抑制剂。CLK1-IN-2 对 Clk1 具有选择性，IC50值为 1.7 nM。CLK1-IN-2 可用于肿瘤、杜氏肌营养不良症和病毒感染如HIV-1和流感的研究。
T83177	Activated A Subunit
	Activated A Subunit 适用于合成促进外显子跳跃的寡聚物偶联体。所合成的偶联体与人类抗肌萎缩蛋白基因内特定的序列配对互补，能够有效促进外显子51的跳跃，为肌萎缩疾病研究提供了一种工具。
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.
T83174	Activated EG3 Tail
	Activated EG3 Tail 适用于合成外显子跳跃寡聚物偶联物。该寡聚物偶联物能与人类抗肌萎缩蛋白基因特定的靶位点匹配，进而诱导外显子 51 的跳跃。此类偶联物对于肌肉萎缩症研究具有重要价值。
T81693	NCP2 Anchor
	NCP2 Anchor用于合成外显子跳跃寡聚物偶联物。它的偶联物与人抗肌萎缩蛋白基因特定靶位点配对互补，从而诱导外显子52的跳跃。该偶联物常应用于肌肉萎缩症的科学研究。
T83173	Activated T Subunit
	Activated T Subunit 用于合成可诱导外显子51跳跃的外显子跳跃寡聚物偶联物。该寡聚物偶联物与人类抗肌萎缩蛋白基因中选定靶位点互补，适用于肌肉萎缩症研究。
T74699	Avacincaptad pegol
	Avacincaptad pegol 是核酸适配体，作为 C5 补体抑制剂，用以减轻炎症导致的视网膜色素上皮 (RPE) 损伤。该化合物可用于研究 stargardt 黄斑营养不良 (STGD1) 与地理萎缩 (GA)。
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...
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。
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。
T38187	6-Aminophenanthridine
	6-Aminophenanthridine is an antiprion agent. It inhibits prion formation in yeast- and mammalian-based screening assays when used alone and, to a greater extent, when used in combination with the α2-adrenergic receptor agonist guanabenz . 6-Aminophenanthridine (300 μM) inhibits protein folding activity of the ribosome (PFAR) by directly competing with protein substrates for the active site and decreases the yield of refolded protein without affecting the refolding rate. It prevents progressive w...

化合物

Cat.No: T39516

Target: PPAR

Cat.No: T7837

Target: Others

Heptamidine dimethanesulfonate

Cat.No: T11553

Synonym: SBi4211 dimethanesulfonate

Target: Others

LM11A-31 dihydrochloride

Cat.No: T11861

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

Neuromuscular-targeting compound 1

Cat.No: T13491

Target: Others

Cat.No: T1805

Synonym: PTC124

Target: CFTR, Autophagy

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: T72073

2-benzylsulfanyl-6-methoxy-4-methylquinazoline

Cat.No: T72072

Target: ROS

Cat.No: T15945

Target: PPAR

CGP 3466B maleate

Cat.No: T21792

Synonym: 马来酸CGP3466B,Omigapil (Maleate),Omigapil maleate

Target: Apoptosis

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: T39517

Synonym: WVE-210201

α7β1 integrin modulator-1

Cat.No: T79918

Target: Integrin

Cat.No: T34426

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

Cat.No: T61182

Cat.No: T11553L

Synonym: SBi4211

Target: Others

Cat.No: T21427

Synonym: Mexitil,Mexiletene,KO-1173,KO1173,KO 1173

Cat.No: T38860

Synonym: Golodirsen,SRP-4053

Cat.No: T66481

Cat.No: T39455

Synonym: NS-065,NCNP-01

Utrophin modulator 1

Cat.No: T61645

Cat.No: T60594

Adenylosuccinic acid

Cat.No: T14129

Synonym: Aspartyl adenylate,Adenylosuccinate

Target: Others

Activated DPG Subunit

Cat.No: T83175

Cat.No: T33595

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

H-D-MeAla-EtVaI-VaI-MeLeu-AIa-D-AIa-MeLeu-MeLeu-MeVaI-MeBmt(OAc)-Abu-O-CH2-CH2-NHMe

Cat.No: T82222

Activated C Subunit

Cat.No: T83176

Cat.No: T73321

Activated A Subunit

Cat.No: T83177

Cat.No: T38859

Synonym: SRP-4045

Activated EG3 Tail

Cat.No: T83174

Cat.No: T81693

Activated T Subunit

Cat.No: T83173

Avacincaptad pegol

Cat.No: T74699

Cat.No: T37449

Adenylosuccinic acid tetraammonium

Cat.No: T73919

Cat.No: T75137

6-Aminophenanthridine

Cat.No: T38187

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