dmd

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。

dmDNA31 is a rifamycin antibiotic that can inhibit the DNA-dependent RNA polymerase of bacteria and has strong bactericidal activity against Staphylococcus aureus.

DMDA-PatA 是一种抑制小分子eIF4A，通过特定方式将eIF4A（一种DEAD-box RNA结合蛋白）紧密夹持于GNG基序，无需ATP即可抑制mRNA的翻译。此化合物在探索癌症及病毒感染的治疗方面显示出潜力。

Idarubicin hydrochloride (Zavedos) 是一种蒽环类抗白血病药物，能抑制细菌和酵母菌生长，还抑制拓扑异构酶 II，干扰 DNA 复制和 RNA 转录。

ABBV-969 Payload(Br-Val-Ala-NH2-bicyclo[1.1.1]pentane-7-MAD-MDCPT)是用于合成ADC 的药物-连接物偶联物，含有Topo I（拓扑异构酶1）抑制分子和连接分子，可以与抗c-Met抗体偶联形成ABBV-969。

p53 and MDM2 proteins-interaction-inhibitor dihydrochloride is a compound that inhibits the interaction between p53 and MDM2 proteins.

The racemic p53 and MDM2 proteins-interaction-inhibitor is an inhibitor of the interaction between p53 and MDM2 proteins.

Mal-Bal-Ph(β-D-Glucose)-7-MAD-MDCPT 为药物-连接子偶联物，主要应用于 ADC 合成。

MP-PEG4-Val-Lys-Gly-7-MAD-MDCPT is a Camptothecin-linker. MP-PEG4-Val-Lys-Gly-7-MAD-MDCPT is a drug-linker conjugate for antibody-drug conjugate (ADC).

7-MAD-MDCPT, a Camptothecin analog, is a toxin payload in antibody drug conjugates (ADCs).

Gly-7-MAD-MDCPT 是一种抗癌剂。Gly-7-MAD-MDCPT 是一种喜树碱 (Camptothecin) 化合物，对多种癌细胞有细胞毒性，IC50为 10-1000 nM。

NH2-bicyclo[1.1.1]pentane-7-MAD-MDCPT(化合物I)作为一种拓扑异构酶I抑制剂，通过偶联抗体实现对细胞的靶向传递，展现出良好的体内外ADC活性。

NH2-bicyclo[1.1.1]pentane-7-MAD-MDCPT（化合物I）盐酸盐是一种通过偶联抗体实现靶向细胞传递的拓扑异构酶I抑制剂，显示出优良的体内外ADC活性。

MP-PEG8-Val-Lys-Gly-7-MAD-MDCPT为喜树碱-接头化合物，属于抗体偶联活性分子（drug-linker conjugate for ADC）部分。该化合物在癌症及自身免疫性疾病研究中显示出研究潜力。

PROTAC(H-PGDS)-7 是一种有选择性的、有效的小分子造血前列腺素 D 合酶 （H-PGDS）PROTAC 降解剂，在 KU812细胞中展现出降解活性，DC50为 17.3 pM。PROTAC(H-PGDS)-7是一种类药化合物，能有效抑制前列腺素 D2 (PGD2)的产生，抑制炎症因子重生作用。PROTAC(H-PGDS)-7是治疗 DMD 和其他H-PGDS 相关疾病的候选化合物。

Adenylosuccinic acid (Adenylosuccinate) 是一种嘌呤核苷酸循环代谢物，是细胞保护剂 Nrf2 的新型诱导剂，可用于研究杜氏肌营养不良症 (DMD) 。

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。

MI-1063 是一种DMD-2抑制剂，能够阻断MDM2与p53的相互作用，从而激活p53的肿瘤抑制功能。MI-1063 对RS4-11和MV4-11癌细胞的生长有抑制效果，其IC50值分别为179 nM和93 nM。MI-1063 还可作为靶蛋白配体用于合成PROTAC降解剂MD-265。

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。

RTC13 是一种通读试剂，可在杜氏肌营养不良症 （DMD） 的 mdx 小鼠模型中恢复抗肌萎缩蛋白表达的能力。

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-167832 exhibits very low MICs against laboratory strains of M. tuberculosis H37Rv (MIC: 0.0005 μg ml) and Kurono (MIC: 0.0005 μg ml) and strains with monoresistance to rifampin (RIF), isoniazid (INH), ethambutol (EMB), streptomycin (STR), and pyrazinamide (PZA) (MIC: 0.00024-0.001 μg ml). However, OPC-167832 has minimal or no activity against standard strains of nonmycobacterial aerobic and anaerobic bacteria[1].The IC90 values of OPC-167832 against intracellular M. tuberculosis strains H37Rv and Kurono are 0.0048 and 0.0027 μg ml, respectively. OPC-167832 shows bactericidal activity against intracellular M. tuberculosis at a low concentration, and the bactericidal activity is saturated at concentrations of 0.004 μg ml or higher[1]. OPC-167832 (oral administration; 0.625-10 mg kg) exhibits a good pharmacokinetic characteristic. The plasma reaches peak at 0.5 h to 1.0 h (tmax) and is eliminated with a half-life (t1 2) of 1.3 h to 2.1 h OPC-167832 distribution in the lungs is approximately 2 times higher than that in plasma, and the Cmax and AUCt of OPC-167832 in plasma and the lungs shows dose dependency[1].OPC-167832 (oral administration; 0.625-10 mg kg; 4 weeks) significantly reduces lung CFU compared to the vehicle group. The dose-dependent decrease of lung CFU is observed from 0.625 mg kg to 2.5 mg kg. In a M. tuberculosis Kurono-infected ICR female mice model. OPC-167832 combines with DMD, BDQ, or LVX via oral gavage exhibits significantly higher efficacies than each single agent alone[1].[1].OPC-167832 (oral gavage; 2.5 mg kg; combination with DCMB; 12 weeks) demonstrates the most potent efficacy when compares with DC, DCB. The lung CFU count after 6 weeks of treatment is below the detection limit, and at the end of just 8 weeks of treatment, the bacteria in the lungs of all the evaluated mice had already been eradicate[1]. [1]. Norimitsu Hariguchi, et al. OPC-167832, a Novel Carbostyril Derivative with Potent Antituberculosis Activity as a DprE1 Inhibitor.Antimicrob Agents Chemother. 2020 May 21;64(6):e02020-19.

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)。

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].

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.