smn

SMN-C3 (MV8T2MCK57) 是口服有效的 SMN2 剪接调节剂。SMN-C3可用于脊椎肌肉萎缩 (SMA) 的研究。

Branaplam (LMI 070) 是一种高效、选择性和口服活性的小分子 SMN2 剪接调节剂，对 SMN 的 EC50为 20 nM。它可提高全长 SMN 蛋白并延长其生存期，也抑制 hERG，IC50为 6.3 μM。

SMN-C2 是一种选择性的 SMN2 基因剪接调节剂，是risdiplam类似物，是调节前 mRNA 剪接的选择性 RNA 结合配体，通过结合 SMN2 pre-mRNA 来发挥作用。SMN-C2 具有研究脊髓性肌萎缩症 (SMA) 的潜力。

SMN2-Stablizer-27 is a stablizer of survival of motor neuron 2 (SMN2), and a post-translationally stablizing SMN protein for the treatment of spinal muscular atrophy (SMA).

RG7800 hydrochloride is an orally active modulator of SMN2 splicing.

SMND-309 is a metabolite of salvianolic acid B. It also shows neuroprotective effects in cultured neurons and in permanent middle cerebral artery occlusion rats.

Risdiplam (RG7916)是一种中枢和外周分布的并可以通过口服给药的小分子 SMN2 前 mRNA 剪接修饰剂，可增加运动神经元存活 (SMN) 蛋白水平。

NVS-SM2 is an effective small molecule splicing enhancer of SMN2 with oral activity. NVS-SM2 showed increased exon 7 inclusion and upregulated SMN protein expression in SMA fibroblasts and SMN cells Δ7 5025 mouse myoblasts.

Branaplam (LMI070; NVS-SM1) hydrochloride 是一种选择性、口服活性的 SMN2 剪接调节剂，能够直接作用于 SMN，其 EC50 值为 20 nM。Branaplam hydrochloride 还可抑制 hERG 通道，其 IC50 值为 6.3 μM。在严重脊髓性肌萎缩症（SMA）小鼠模型中，Branaplam hydrochloride 显著增加了全长 SMN 蛋白水平，并明显延长了生存期。

BIIE-0246 is a potent and highly selective non-peptide neuropeptide Y (NPY) Y2 receptor antagonist. With an IC50 of 15 nM.

CP-105696 是一种具有强效性和选择性白三烯 B4 (LTB4) 受体拮抗剂，可用于研究异体移植物排斥反应。

HJB97 可用于开发设计 PROTAC BET 的降解剂，具有抗肿瘤活性。HJB97 是高亲和力的 BET 抑制剂，Ki 值分别为 0.9 nM (BRD2 BD1)，0.27 nM (BRD2 BD2)，0.18 nM (BRD3 BD1)，0.21 nM (BRD3 BD2)，0.5 nM (BRD4 BD1)，1.0 nM (BRD4 BD2)。

Toreforant is an effective and selective histamine H4 receptor antagonist (Ki at the human receptor: 8.4 nM).

WAY 163909 is an effective and selective agonist of the 5-HT(2C) receptor (Ki: 10.5±1.1 nM).

Branaplam is a highly potent, selective and orally active SMN2 splicing modulator. Branaplam increases the amount of functional SMN protein produced by the SMN2 gene through modifying its splicing.

ML372 is a potent and selective SMN Modulator (EC50 = 12 nM, 325% increase inSMN2) with good potency, pharmacokinetics, tolerance, and CNS penetration.

Rg3039 (PF-06687859) 是一种具有口服活性、能够透过血脑屏障的DcpS 抑制剂，其IC50=0.069 nM。

The Survival of Motor Neurons (SMN) protein participates in RNA splicing. Decreases in SMN, typically a consequence of defects in the smn1 gene, result in the death of motor neurons and lead to the neurodegenerative disease, spinal muscular atrophy (SMA). Cuspin-1 is a small molecule upregulator of SMN that has been shown in vitro to increase levels of SMN in SMA patient fibroblasts by 50% at 18 μM. Its mechanism of action is thought to involve increased phosphorylation of ERK to initiate Ras-Raf-MEK signaling, which results in an increased rate of SMN translation.

Nusinersen（诺西那生）是一种用于治疗小儿和成人脊髓性肌萎缩症（SMA）的反义寡核苷酸（ASO），改变SMN2基因的加工方式并增加SMA患者缺乏的SMN蛋白的产生。

RG7800 hydrochloride 是一种口服具有活力的 SMN2 剪接调节剂，能够作用于 SMN2 剪接 (EC1.5x: 23 nM) 及 SMN 蛋白 (EC1.5x: 87 nM)。

RG7800 hydrochloride为一可口服SMN2剪接调节剂，其对SMN2剪接及SMN蛋白的EC1.5x值分别为23 nM及87 nM。